Program Book

2021 VIRTUAL NF CONFERENCE JUNE 14 - 16, 2021

KEEPING THE NF COMMUNITY CONNECTED

CONTENTS Table of Contents

Welcome Message...... 4

Foundation Staff...... 5

Advisory Boards...... 6

CTF Europe...... 7

Funding Opportunities...... 8

The Friedrich von Recklinghausen Award: NF Tradition and Progress...... 10

Schedule at a Glance...... 11

Conference Co-Chairs...... 12

Agenda...... 13

Participants...... 18

Other Programs...... 26

Acknowledgments...... 32

Sponsors and Supporters...... 33

2021 NF Virtual Conference · June 14-16, 2021 | 3 Dear NF Conference Attendees,

I want to start this letter a little differently than usual: I want to say THANK YOU! Even if COVID-19 challenged us on every level, this NF community has remained together in a way that I would have never imagined. That is because of all of you: researchers, clinicians, patients, donors, volunteers, board and staff, staying true to our shared mission, all worked around the clock!

Although we were initially hoping to hold the 2021 NF Conference in person this year, the pandemic has forced us to go virtual once again. Our great thanks to Drs. Laura Papi and Thomas De Raedt, who accepted the baton from last year’s co-chairs who built the base for this year’s meeting, and agreed to transition this virtual meeting to next year’s in-person event. Altogether, this collaborative team has successfully put together a program that is top notch, enriching and inspiring.

This year’s conference will again focus on giving the floor to the new and exciting discoveries. Platform presentations covering all forms of NF are a nice mix of basic, translational, and clinical science, carefully selected by the reviewers. Moreover, all posters will be available as e-posters, so there will be ample opportunities to interact.

At the Children’s Tumor Foundation our work has had an increasingly global reach. In February, CTF hosted an educational seminar for Latin American clinicians with over 550 registrants. In Europe, our activities continue to grow, including the development of platform trials for NF and the planning of Master Classes by the European Clinical Care Advisory Board.

The USA CCAB continues to inspire the community with their launch of so many important programs such as the virtual case conferences and the project focusing on understanding access and barriers to new treatments.

This year, we are holding our annual Hackathon again (year 3) and we expanded from Hack for NF to Hack for Rare. The kick-off will be later this month, with participants joining us from around the world.

Major breakthroughs continue within our community: May marked the publication of the updated diagnostic criteria for NF1 and Legius syndrome with the manuscript of the diagnostic criteria for NF2 and schwannomatosis being prepared for submission for publication. While we are still celebrating the first-ever drug approval for NF1 in the USA, the drug is now approved in Brazil, South Korea and Europe; the first-ever platform trial for NF2 has fully recruited all patients, and the first-ever clinical trial for schwannomatosis is underway.

This conference is again filled with news of remarkable advancements in care and towards a cure. It is truly an honor to bring the work of this amazing community together, even if virtually. Next year we will gather in person, but until then, ever-onward, united in our efforts to end NF!

Be safe, be well and we look forward to lots of hugs next year in Philadephia!

Warm regards,

ANNETTE BAKKER President

4 | Children’s Tumor Foundation · Ending Neurofibromatosis Through Research FOUNDATION STAFF Children’s Tumor Foundation

Annette Bakker, PhD - President CTF BOARD OF DIRECTORS Danielle Baker - Director, Human Resources Elizabeth Oliver - Senior Executive Assistant Executive Members Tracy Galloway - Chair Research and Medical Programs Gabriel Groisman - Vice Chair Salvatore La Rosa, PhD - Chief Scientific Officer RB Harrison - Secretary Vidya Browder, PhD - Basic Science Senior Manager Randall Stanicky - Treasurer Kate Kelts - Patient Support Coordinator Pamela Knight - Senior Director, Clinical Program Board Members Daniel Altman Patrice Pancza - Vice President, External Relations Amy Boulas Heather Radtke - NF Clinic Network Senior Manager Robert Brainin Traceann Rose - Director, Patient and Volunteer Engagement D. Wade Clapp Daniel Gilbert Development John Golfinos Michele Przypyszny - Chief Advancement Officer Anita Gribben Claudia Becerra - Manager, Development Carol Harrison Kalagher Allison Cote - Senior Manager, Donor Relations Frank Haughton Emily Crabtree - Senior Director Development Operations Richard Horvitz Allyson Douglass - Manager, Special Events Bruce Korf Angela Dumadag - Senior Manager, Development Simone Manso Barbara Gallagher - Vice President, Development Steven L. McKenzie Julie Nassisi - Senior Manager, Youth Programs Laura Perfetti Kim Robinson - Manager, Special Events Michael Peterson Connie Sorman - Senior Manager, Stewardship & Volunteer Development Scott Plotkin Rebecca Taylor - Senior Manager, Development Liz Rodbell Kelly Vigil - Endurance Coordinator Kenneth Rudd Richard Soll Jennifer Tezbir - Development Manager, Walk Stuart Suna

Finance and Administration Advisory Board Members Sarah Bourne - VP Finance & Operations Bruce R. Korf, MD, PhD - Chair, Medical Advisory Jennifer Truong - Gift Processing Coordinator Scott Plotkin, MD, PhD - Chair, Clinical Care Advisory Board Monique Boucher - Gift Processing Senior Manager D. Wade Clapp, MD - Chair, Research Advisory Board Carey Milligan - Senior Accounting Manager Brandon Weaver-Bey - Gift Processing Coordinator Volunteer Leadership Council Chair Anita Gribben Marketing and Communications Renie Moss Simon Vukelj - Marketing Officer Rebecca Harris - Public Relations Senior Manager Honorary Directors Alissa Marks - Director Marketing Suzanne Earle Susanne Preinfalk - Design Director Linda Halliday Martin Vanessa Younger - Communications Director Carolyn E. Setlow Edward M. Stern - Pro Bono Counsel Nate Walker Allan E. Rubenstein, MD - Director of Medical Affairs, Emeritus

2021 NF Virtual Conference · June 14-16, 2021 | 5 ADVISORY BOARDS Children’s Tumor Foundation Advisory Boards

BUSINESS ADVISORY COUNCIL CLINICAL CARE ADVISORY BOARD

Randall Stanicky (Co-Chair) Scott Plotkin, MD, PhD, Massachusetts General Hospital Alex Zisson (Co-Chair) Council Members Council Members Oliver F. Adunka, MD, FACS, The Ohio State University Anna Barker, PhD Jaishri Blakeley, MD, Johns Hopkins University Rob Brainin Gary Brenner, MD, PhD, Massachusetts General Hospital Stephen Friend, PhD Jan Friedman, MD, PhD, University of British Columbia Stephen Groft, PharmD Jennifer Janusz, PsyD, ABPP-CN, Children’s Hospital Colorado Edward Stern Justin Jordan, MD, MPH, Massachusetts General Hospital Vicki Match Suna, AIA Bonnie Klein-Tasman, PhD, University of Wisconsin-Milwaukee Daniela Brunner, PhD Laura Klesse, MD, PhD, UT Southwestern Medical Center Tena Rosser, MD, Children’s Hospital of Los Angeles MEDICAL ADVISORY COMMITTEE Pamela Trapane, MD, MME, UF Health Jacksonville Nicole Ullrich, MD, PhD, Boston Children’s Hospital Bruce Korf, MD, PhD (Chair), University of Alabama at Birmingham David Viskochil, MD, PhD, Chair Emeritus; University of Utah Kaleb Yohay, MD, NYU Langone Medical Center Committee Members Peggy Wallace, PhD, University of Florida Board Liaisons Andrea McClatchey, PhD, Massachusetts General Hospital Steve McKenzie Michael Fisher, MD (Chair of DoD NF Clinical Trial Consortium), Michael Peterson Children’s Hospital of Philadelphia Richard Soll Jaishri Blakeley, MD, Johns Hopkins University, Director of NF Tracy Galloway Therapeutic Acceleration Program David Miller, MD, PhD, Boston Children’s Hospital, Scientific Director Patient Representatives of the Neurofibromatosis Research Initiative (NFRI) Alwyn Dias, MSW, ACC - NF1 YooRi Kim, MS, Scientific Officer, Gilbert Family Foundation Reema Bogin - NF2 Scott Plotkin, MD, PhD, Massachusetts General Hospital Dale Berg - Schwannomatosis Nader Futouhi, PhD, Chief Scientific Officer - TB Alliance George C. Terstappen, PhD, Academic Visitor, University of Oxford, CTF Staff Department of Pharmacology Pamela Knight, MS, Director, Clinical Program D. Wade Clapp, MD, Indiana University Heather Radtke, MS, CGC, Manager, NF Clinic Network Brigitte Widemann, MD, National Cancer Institute David Viskochil, MD, PhD, University of Utah Marco Nievo, PhD, Chief Scientific Officer, CTF Europe

Board Liaisons Richard Soll Gabe Groisman Rob Brainin Tracy Galloway Richard Horvitz

CTF Staff Annette Bakker, PhD, President Salvatore La Rosa, PhD, Chief Scientific Officer

6 | Children’s Tumor Foundation · Ending Neurofibromatosis Through Research CTF EUROPE Children’s Tumor Foundation Europe

CTF EUROPE BOARD OF DIRECTORS CLINICAL CARE ADVISORY BOARD - EUROPE

Simone Manso (Chair) Pierre Wolkenstein, MD, PhD (Chair) Annette Bakker, PhD (Vice Chair) Hopital Henri-Mondor, Paris, France Rosalie Ferner, MD (Chair) Directors King’s College, London, UK Magda Chlebus Stuart Suna Council Members Nikola Gazdov Amedeo Azizi, MD, PhD, Medical University of Vienna, Austria Richard Horvitz Ignacio Blanco, MD, PhD, Germans Trias Hospital, Barcelona, Spain Gareth Evans, MD, University of Manchester Said Farschtschi, MD, University Medical Center Hamburg-Eppendorf, Germany Shruti Garg, MBBS, MRCPSYCH, MMEDSCI, PhD, University of Manchester & Royal Manchester Children’s Hospital Michel Kalamarides, MD, PhD, Hopital Pitie-Salpetriere Hospital, Sorbonne University, Paris, France Eric Legius, MD, PhD, University Hospital of Leuven, Belgium Viktor-Felix Mautner, MD, PhD, University Medical Center Hamburg- Eppendorf, Germany Rianne Oostenbrink, MD, PhD, Erasmus MC, Rotterdam, The Netherlands Laura Papi, MD, PhD, University of Florence, Firenze, Italy Juha Peltonen, MD, PhD, University of Turku, Finland Sirkku Peltonen, MD, PhD, University of Gothenburg, Sweden Scott Plotkin, MD, PhD, Chair CCAB-US, Massachusetts General Hospital, Boston, Massachusetts Cornelia Potratz, MD, University Medicine Berlin, Germany Cecilie Rustad, MD, Oslo University Hospital, Norway Eva Trevisson, MD, PhD, University Hospital of Padova, Italy

2021 NF Virtual Conference · June 14-16, 2021 | 7 CHILDREN’S TUMOR FOUNDATION FUNDING OPPORTUNITIES

THE DISCOVERY FUND

The Children’s Tumor Foundation is dedicated to funding research that will prevent, reverse, or manage the complications of NF1, NF2, and schwannomatosis, and to improve the quality of life of those affected with these disorders. As the leading non-governmental NF research funder in the US, CTF has acted as a ‘seed funder’ by providing funding especially to early-career NF researchers and to early-stage NF research, both of which require additional time and support before becoming eligible to apply for larger governmental or industrial funding.

To facilitate the advancement of NF research and to better serve the NF community, CTF has established the Discovery Fund as a mechanism to support researchers and clinicians engaged in novel and fundamental NF investigations.

YOUNG INVESTIGATOR AWARD (YIA)

CTF’s longest-running competitive award program since the mid-1980s, the YIA has traditionally provided two years of funding to early-career NF researchers to enable these trainees become independent investigators in NF-related fields.

AWARD AMOUNT: $64,000 to $174,000 AWARD DURATION: 2 to 3 years

Dates to note for upcoming 2022 YIA cycle:

• Monday, December 6, 2021 - YIA Letter of Intent (LOI) form goes live • Monday, January 3, 2022 - YIA LOIs due by 5:00 pm EST • Friday, January 21, 2022 - YIA invitation to full application • Friday, March 4, 2022 - YIA full applications due by 5:00 pm EST • Week of May 20, 2022 - YIA decision notifications

For 2022, CTF will provide postdoctoral YIA salary support for up to THREE years. The third year of funding will be contingent upon satisfactory progress of the first two years. Rates are commensurate with experience.

Please visit https://www.ctf.org/research/young-investigator-award-yia for more details. We look forward to receiving your applications!

8 | Children’s Tumor Foundation · Ending Neurofibromatosis Through Research CHILDREN’S TUMOR FOUNDATION FUNDING OPPORTUNITIES

DRUG DISCOVERY INITIATIVE-REGISTERED REPORTS (DDI-RR)

The Children’s Tumor Foundation (CTF) and the scientific journal PLOS ONE in 2017 launched a new funding program in the area of neurofibromatosis (NF) research based on the Registered Reports model. The new initiative, called the Drug Discovery Initiative Registered Report (DDIRR) Awards, is a funder-publisher partnership that integrates the Registered Reports model in the grant application process. This model will allow for more rigorous, reproducible and transparent science, guaranteeing its awardees with an in-principle acceptance (IPA) option to publish in the journal PLOS ONE, regardless of study outcome. DDIRR recipients can submit their research to any journal.

AWARD AMOUNTS: up to $40,000 for in-vitro studies (including indirect costs) up to $85,000 for in-vivo studies (including indirect costs)

Dates to note for upcoming DDIRR cycle:

• Monday, September, 2021 - DDIRR Letter of Intent (LOI) form live • Monday, October 18, 2021 - DDIRR LOI due by 5:00 pm EST • Monday, November 1, 2021 - DDIRR invitation to full application • Monday, November 29, 2021 - DDIRR full proposals due by 5:00 pm EST • December 2021 - DDIRR decision notifications Clinical Research Awards (CRA)

Please visit https://www.ctf.org/research/drug-discovery-initiative-registered-reports-ddirr for more details.

CLINICAL RESEARCH AWARD (CRA)

The CRA program supports research that may lead to therapies for neurofibromatosis type 1 (NF1), neurofibromatosis type 2 (NF2), and/or schwannomatosis. Funding proposals may include treatment studies as well as clinical trial enabling studies of pharmacologic and physical therapies, surgical innovations, diagnostics and biomarkers, and neurocognitive or psychosocial interventions. Proposals should present broad-thinking, novel ideas.

AWARD AMOUNTS: Applicants may propose a budget of up to $150,000 (including indirect costs).

Dates to note for upcoming CRA cycle:

• Monday Aug 16, 2021 – CRA Letter of Intent (LOI) form launched • Monday Sept 13, 2021 - CRA LOI’s due by 5:00 pm EST • Monday Sept 27, 2021 - CRA invitation to full application • Monday Oct 25, 2021 - Full applications due by 5:00 pm EST • Week of Dec 13, 2021 - CRA decision notifications

Please visit https://www.ctf.org/research/clinical-research-award-cra for more details.

2021 NF Virtual Conference · June 14-16, 2021 | 9 The Friedrich von Recklinghausen Award: Neurofibromatosis Tradition and Progress

The Children’s Tumor Foundation’s Friedrich von Recklinghausen Award is given to individuals in the professional neurofibromatosis community who have made significant contributions to neurofibromatosis research or clinical care. It is named after Friedrich Daniel von Recklinghausen (1833-1910), the German physician who first described ‘von Recklinghausen’s disease’ – what we now know as neurofibromatosis type 1.

2021 Friedrich von Recklinghausen Award Recipient

With great pleasure, the Children’s Tumor Foundation announces the 2021 recipient of the Friedrich von Recklinghausen Award, Prof. Dr. Marco Giovannini (MD, PhD) of the University of California, Los Angeles (UCLA). He is Professor-in-Residence, Head and Neck Surgery at UCLA, Director of the Neural Tumor Research Laboratory, and member of the Signal Transduction and Therapeutics Program at the UCLA Jonsson Comprehensive Cancer Center. He is also is co-director of the UCLA Multidisciplinary NF Clinic.

As a researcher, Dr. Giovanini has a long-term interest in the genetics and biology of human cancer predisposition and progression. His lab has made significant contributions to the characterization of many major NF tumor suppressors especially NF2, and SMARCB1. The overarching goal of his lab is to effectively translate the discoveries into medicines that will improve the quality and long-term survival of NF2 and schwannomatosis patients. He recognized that the lack of preclinical NF2 and schwannomatosis models present a real roadblock to developing better treatments for patients.

Not only did his lab develop a variety of innovative cell and animal models that faithfully replicate human tumorigenesis, but he is also known to openly share the models with the community and test novel discovery paradigms swiftly.

He is creative, always open to collaborating, and is now in his third year (2019, 2020, 2021) acting as the NF2/ Schwannomatosis mentor to the hackathon participants. This is emblematic of his enthusiasm to share his vast knowledge, not just within the community, but outside of NF to help educate and raise awareness.

We are very proud and delighted to recognize Dr. Giovannini’s significant achievements with the 2021 Friedrich von Recklinghausen Award. Please join us in congratulating Dr. Giovannini for this well-deserved honor.

The following are the most recent recipients of the Award:

2008 2009 2010 2012 2013 2014 Vincent ‘Vic’ Riccardi, MD Luis Parada, PhD Nancy Ratner, PhD David Gutmann, MD, PhD Brigitte Widemann, MD Gareth Evans, MD The Neurofibromatosis Institute University of Texas Cincinnati Children’s Hospital Washington University National Cancer Institute St. Mary’s Hospital, Southwestern Medical Center University of Manchester, UK

2015 2016 2017 2018 2019 2020 Eric Legius, MD, PhD David Viskochil, MD, PhD Karen Cichowski, PhD Ludwine Messiaen, PhD Scott Plotkin, MD, PhD D. Wade Clapp, MD University of Leuven, Belgium University of Utah Harvard Medical School University of Alabama Massachusetts General Hospital Indiana University at Birmingham Harvard Medical School School of Medicine

10 | Children’s Tumor Foundation · Ending Neurofibromatosis Through Research SCHEDULE Schedule At-A-Glance TIME EVENT 9:15 AM 9:30 AM Welcome 9:30 AM 12:00 PM CLINICAL CARE SYMPOSIUM

12:00 PM 12:30 PM Break 12:30 PM 1:00 PM Mission Moment 1:00 PM 2:30 PM Tumorigenesis JUNE 14 MONDAY 2:30 PM 2:45 PM Break 2:45 PM 4:15 PM NF2/Schwannomatosis: Bench To Bedside – Concurrent Session 2:45 PM 4:15 PM Immunotherapy and the Microenvironment – Concurrent Session 8:25 AM 9:25 AM Satellite – MEK Inhibitors and NF1-Associated Tumors: Molecular Mechanisms and Targeted Approaches 9:30 AM 10:45 AM Pain 10:45 AM 10:50 AM Break

10:50 AM 12:05 PM Gene Therapy 12:05 PM 12:30 PM Break 12:30 PM 2:00 PM New Investigations in Cognitive and Psychosocial Research for Neurofibromatosis – Concurrent Session JUNE 15 TUESDAY 12:30 PM 2:00 PM NF2/Schwannomatosis Clinical Care – Concurrent Session 2:00 PM 2:15 PM Break 2:15 PM 4:15 PM PLATFORM PRESENTATIONS – BASIC SCIENCE 2:15 PM 4:15 PM PLATFORM PRESENTATIONS – CLINICAL SCIENCE 8:30 AM 9:30 AM European Initiatives – Update (Non-CE Activity) 9:30 AM 10:00 AM Poster Competition Winners – Short Oral Presentations

10:00 AM 11:30 AM Consortia Updates 11:30 AM 11:45 AM Break 11:45 AM 12:15 PM Late Breaking Abstracts – Platform Presentation

JUNE 16 12:15 PM 12:30 PM Hackathon – From Hack for NF to Hack4Rare (Non-CE Activity)

WEDNESDAY 12:30 PM 12:50 PM Presentation of 2021 Friedrich Von Recklinghausen Award 12:50 PM 1:05 PM Wrap-Up and Adjournment 1:15 PM 5:00 PM REiNS Meeting

THE MEETING TIMES LISTED ARE EASTERN DAYLIGHT TIME

2021 NF Virtual Conference · June 14-16, 2021 | 11 BIOS 2021–2022 NF Conference Co-Chairs

Thomas De Raedt, PhD, Children’s Hospital of Philadelphia

Dr. De Raedt researches pediatric high grade glioma development and aims to understand the involvement of crucial pathways. He investigates pathway interaction, and explores ways to develop therapies through analyzing human tumors, performing cellular studies, and developing accurate mouse models. This allows Dr. De Raedt and his team to perform novel pre-clinical studies that can lead to clinical trials.

Laura Papi, MD, University of Florence, Italy

Dr. Laura Papi is Associate Professor at the Department of Clinical Physiopathology, Medical Genetics Unit at the University of Florence, Italy. Her research areas are in molecular and genetic studies in Neurofibromatosis type 2 and schwannomatosis, and molecular and epidemiological studies on Hereditary breast-ovarian cancer syndrome.

2020 NF Conference Co-Chairs

Matthias Karajannis, MD, MS Conxi Lázaro, PhD Nicole J Ullrich, MD, PhD Chief of the Pediatric Neuro-Oncology Service, Head of the Molecular Diagnostic Unit, Associate Professor of Neurology, Memorial Sloan Kettering Cancer Center Hereditary Cancer Program, Catalan Institute Boston Children’s Hospital of Oncology (ICO-IDIBELL), Barcelona, Spain

12 | Children’s Tumor Foundation · Ending Neurofibromatosis Through Research AGENDA Monday · June 14, 2021 (All times are ET)

9:15 AM 9:30 AM WELCOME

Thomas DeRaedt, PhD, Children’s Hospital of Philadelphia; Laura Papi, MD, University of Florence, Italy; Annette Bakker, PhD, President, Children’s Tumor Foundation 9:30 AM 12:00 PM CLINICAL CARE SYMPOSIUM Symposium Chair: Nicole Ullrich, MD, PhD, Boston Children’s Hospital

Symposium Introduction and CTF Clinical Care Advisory Boards 9:30 AM 9:40 AM CCAB-US: Scott Plotkin, MD, PhD, Massachusetts General Hospital CCAB-Europe: Pierre Wolkenstein, MD, PhD, Hopital Henri-Mondor, Creteil, FR

CCAB US Mission and Objectives 9:40 AM 9:50 AM Scott Plotkin, MD, PhD

Access to NF Clinics and Receipt of Guideline Concordant NF Care in the U.S. 9:50 AM 10:10 AM Vanessa Merker, PhD, Massachusetts General Hospital

Health Care Transition in the Neurofibromatoses 10:10 AM 10:30 AM Tena Rosser, MD, Children’s Hospital Los Angeles; David Viskochil, MD, PhD, University of Utah

Patient Access to Novel NF Treatments – Current Clinical Practices and Identification of Barriers 10:30 AM 10:50 AM Laura Klesse, MD, PhD, UT Southwestern

10:50 AM 11:00 AM BREAK

Virtual Case Discussion: NF1 Patient with Complex Medical and Psychiatric Issues Moderator: Nicole Ullrich, MD, PhD, Boston Children’s Hospital 11:00 AM 12:00 PM Case Presenter: Pamela Trapane, MD, Universisty of Florida - Jacksonville Health Expert Panel: David Viskochil, MD, PhD; Laura Klesse, MD, PhD; Rosalie Ferner, MD, FRCP, Guy’s and St. Thomas’ Hospital, NHS Trust, UK

12:00 PM 12:30 PM BREAK 12:30 PM 1:00 PM MISSION MOMENT

The Patient Journey Moderator: Traceann Rose, Director, Patient Engagement - CTF Patient Participants: Dale Berg, Lilly Ann Brooks, Alwyn Dias, Cole Harrell, William Riter

1:00 PM 2:30 PM TUMORIGENESIS

Session Chairs: Ping Chi, PhD, Memorial Sloan Kettering Cancer Center; Angela Hirbe, MD, PhD, Washington University

Novel Therapeutic Strategies for MPNST 1:00 PM 1:25 PM Christine Pratilas, MD, Johns Hopkins University

Platform Talk: A Case-Only Study in 1,333 NF1 Patients to Identify Genetic Modifiers of Cutaneous, Subcutaneous and 1:25 PM 1:38 PM Plexiform Neurofibromas Laurence Pacot, Cochin Institute, France

Platform Talk: T-Cells are Essential for Plexiform Neurofibroma Growth 1:38 PM 1:51 PM Jay Pundavela, PhD, Cincinnati Children’s Hospital

hiPSC and Murine Models of Plexiform Neurofibromas and MPNST 1:51 PM 2:16 PM Lu Le, MD, PhD, UT Southwestern

Platform Talk: NF2-TEAD Autopalmitoylation Inhibitors Slow Tumor Cell Proliferation in the Pre-Clinical Postn-Cre 2:16 PM 2:30 PM Schwannomam in vivo Model Liyam Laraba, PhD, University of Plymouth, UK 2:30 PM 2:45 PM BREAK

2021 NF Virtual Conference · June 14-16, 2021 | 13 AGENDA Monday · June 14, 2021 (All times are ET) 2:45 PM 4:15 PM NF2/SCHWANNOMATOSIS: BENCH TO BEDSIDE – CONCURRENT SESSION Session Chairs: Matthias Karajannis, MD, MS, Memorial Sloan Kettering Cancer Center; Miriam Smith, PhD, University of Manchester, UK

LZTR1: Its Relation to Schwannomatosis 2:45 PM 3:10 PM Anna Sablina, PhD, KU Leuven, Belgium

Meet DGCR8 in the Middle 3:10 PM 3:35 PM Barbara Rivera Polo, PhD, IDIBELL, Spain Platform Talk: Preclinical Assessment of Fimepinostat, a Dual Phophpoinositide-3 Kinase/Histone Deacetylases 3:35 PM 3:55 PM Inhibitor as a Therapeutic for NF2 Schwannomas Maria Martinelli, University of Central Florida

Platform Talk: Pre-Clinical Assessment of Combinatorial PI3K/mTOR and FAK/SRC Pathway Inhibitors for Treatment of 3:55 PM 4:10 PM NF2 Schwannomas Haley Hardin, University of Central Florida 2:45 PM 4:15 PM IMMUNOTHERAPY AND THE MICROENVIRONMENT – CONCURRENT SESSION Session Chairs: David Gutmann, MD, PhD, Washington University, St. Louis; Antonio Iavarone, MD, Columbia University

Immunology of the Brain Tumor Microenvironment 2:45 PM 3:10 PM David Gutmann, MD, PhD, Washington University, St. Louis

Immunology of the Peripheral Nerve Sheath Tumor Microenvironment 3:10 PM 3:35 PM Steven Rhodes, PhD, Indiana University

State of Immune System in GBM – Implication for Immune Therapy Trials 3:35 PM 4:00 PM David Reardon, MD, Dana Farber Cancer Institute/Harvard University

Platform Talk: Epigenetic Reprogramming Drives Schwann Cell Transformation and Treatment Resistance in Malignant 4:00 PM 4:15 PM Peripheral Nerve Sheath Tumors (MPNSTs) Harish Vasudevan, MD, PhD, University of California San Francisco Tuesday · June 15, 2021 (All times are ET) SATELLITE – MEK INHIBITORS AND NF1-ASSOCIATED TUMORS: MOLECULAR MECHANISMS 8:25 AM 9:25 AM AND TARGETED APPROACHES Translating Science to Transform Clinical Practice How MEK Inhibitors are Changing the Treatment Landscape of NF1 and NF1-Associated Tumors Website: PeerView.com/LiveNF21-Virtual Neurofibromatosis type 1 (NF1) is a common inherited disorder with clinical heterogeneity and high predisposition for developing tumors. While these tumors are generally benign, they may cause health problems by pressing on nearby body tissue. For early diagnosis and optimal care, clinicians must be aware of the clinical features of NF1 and the available therapeutic options. This PeerView MasterClass and Practicum live web broadcast will aid in the diagnosis and monitoring of NF1-associated complications by reviewing the role of MEK inhibitors for treating tumors, and discussing current and emerging therapeutic options for the treatment of plexiform neurofibromas and low-grade gliomas. After each presentation, the faculty will provide guidance on integrating the evidence into everyday patient care. Faculty: Bruce R. Korf, MD, PhD, UAB School of Medicine University of Alabama at Birmingham; Nicole Ullrich, MD, PhD, Boston Children’s Hospital

9:30 AM 10:45 AM PAIN

Session Chairs: Rajesh Khanna, PhD, University of Arizona; Staci Martin, PhD, National Institutes of Health (NIH) Acceptance and Commitment Therapy and Heart Rate Variability: Implications for Pain Management in NF1 9:30 AM 9:50 AM Taryn Allen, PhD, National Cancer Institute (NIH) Distinct Categories and Mechanisms of Pain in NF1, NF2 and Schwannomatosis 9:50 AM 10:10 AM Larry Sherman, PhD, Oregon Health & Science University Genotype and Whole Body MRI Metrics in Schwannomatosis 10:10 AM 10:30 AM Justin T. Jordan, MD, MPH, Massachusetts General Hospital/Harvard Medical School Platform Talk: A Qualitative Understanding of Pain Interests Across Medical Experts and Individuals with NF1 10:30 AM 10:45 AM Frank Buono, PhD, Yale University

14 | Children’s Tumor Foundation · Ending Neurofibromatosis Through Research AGENDA Tuesday · June 15, 2021 (All times are ET)

10:45 AM 10:50 AM BREAK

10:50 AM 12:05 PM GENE THERAPY Session Chairs: Elisabeth Castellanos, PhD, Hospital Germans Trias & Pujol, ESP; Eric Pasmant, PhD, Université de Paris, FR Non-Viral Gene Therapy for NF1 10:50 AM 11:15 AM Jiangbing Zhou, PhD, Yale School of Medicine Platform Talk: Development of Novel Clinically Relevant AAV Vectors for Safe and Efficient Targeting of Human 11:15 AM 11:30 AM Primary Schwann Cells Matthieu Drouyer, PhD, Chldren’s Medical Research Institute, Australia Platform Talk: Developing CRISPR-Based Gene Therapies for NF1 11:30 AM 11:45 AM Stephanie Bouley, PhD, Massachusetts General Hospital

Platform Talk: Testing the Use of Antisense Oligonucleotides to Module the Effect of Splicing and Nonsense Genetic 11:45 AM 12:00 PM Variants Causing NF2 Nuria Catasus, MSc, German Trias I Pujol Research Institute, Barcelona, ESP

12:00 PM 12:05 PM Discussion

12:05 PM 12:30 PM BREAK

NEW INVESTIGATIONS IN COGNITIVE AND PSYCHOSOCIAL RESEARCH FOR 12:30 PM 2:00 PM NEUROFIBROMATOSIS – CONCURRENT SESSION

Session Chairs: Karin Walsh, PsyD, Children’s National Medical Center; Pam Wolters, PhD, National Institutes of Health (NIH) Adolescents and Young Adults (AYA) with Neurofibromatosis Type 1 (NF1): A Descriptive Study of Adaptive Functioning 12:30 PM 12:55 PM Kari Struemph, PhD, University of Kansas School of Medicine Platform Talk: Motor Development in Early Childhood: An Investigation with Young Children with Neurofibromatosis 12:55 PM 1:10 PM Type 1 Sara Pardej, MS, University of Wisconsin-Milwaukee

Platform Talk: Examining Cognitive Load in Children with Neurofibromatosis Type 1 Compared to Typically Developing 1:10 PM 1:25 PM Children Sara Silber, BA, Franklin and Marshall College

Platform Talk: Predictors of Socioemotional Development in Children with Neurofibromatosis Type 1 and Plexiform 1:25 PM 1:40 PM Neurofibromas Yang Hou, PhD, University of Kentucky

Platform Talk: Perspectives of Adults with Neurofibromatosis Regarding the Design of Psychosocial Trials: 1:40 PM 1:55 PM Results from an Anonymous Survey Online Pam Wolters, PhD, National Cancer Institute (NCI) 12:30 PM 2:00 PM NF2/SCHWANNOMATOSIS CLINICAL CARE – CONCURRENT SESSION Session Chairs: Justin Jordan, MD, MPH, Massachusetts General Hospital; Michel Kalamarides, MD, PhD, Hôpital Universitaire Pitié-Salpêtrière, Paris, FR Update on Clinical Trials in NF2 and Schwannomatosis 12:30 PM 12:55 PM Scott Plotkin, MD, PhD, Massachusetts General Hospital/Harvard Medical School Platform Talk: Genetic Testing to Gain Diagnostic Clarity in Neurofibromatosis Type 2 and Schwannomatosis 12:55 PM 1:10 PM Rebecca Burns, BS, MGH Institute of Health Professionals Platform Talk: Assessment of Multiple Meningiomas as a Major Criterion in Neurofibromatosis Type 2 Criteria 1:10 PM 1:25 PM Gareth Evans, MD, University of Manchester, UK Platform Talk: Phase II Study of Axitinib in Patients with Neurofibromatosis Type 2 and Progressive Vestibular 1:25 PM 1:40 PM Schwannomas Sheetal Phadnis, MD, University of Alabama at Birmingham Platform Talk: Longitudinal Evaluation of Pain in Schwannomatosis Patients 1:40 PM 1:55 PM Naomi Askenazi, Massachusetts General Hospital

2:00 PM 2:15 PM BREAK

2021 NF Virtual Conference · June 14-16, 2021 | 15 AGENDA Tuesday · June 15, 2021 (All times are ET) 2:15 PM 4:15 PM PLATFORM PRESENTATIONS – BASIC SCIENCE

Session Chairs: Rebecca Dodd, PhD, University of Iowa; Helen Morrison, PhD, Friedrich Schiller University, Jena, Germany

Complimenting MEK Inhibition in NF1: C4aR-Antagonism 2:15 PM 2:30 PM Melissa Perrino, MD, Cincinnati Children’s Hospital

Proteins Secreted by Painful Schwannomatosis-Related Tumors 2:30 PM 2:45 PM Kimberly Ostrow, PhD, Johns Hopkins University

NF1 Mutation Drives Neuronal Activity-Dependent Optic Glioma Initiation 2:45 PM 3:00 PM Yuan Pan, PhD, Stanford University

Reduced Risk of Schwannoma in 22q11.2 Deletion Syndrome 3:00 PM 3:15 PM Miriam Smith, PhD, University of Manchester, UK

Generation of iPSC-Derived Human Neurofibroma-Like Tumors in Mice 3:15 PM 3:30 PM Helena Mazuelas, PhD, German Trias i Pujol Research Institute, Spain

Losarten Prevents Tumor-Induced Hearing Loss 3:30 PM 3:45 PM Lei Xu, MD, PhD, Massachusetts General Hospital/Harvard University

Identifying Novel Drugs to Treat NF1 Tumors Using Genetic Screens in Drosophila Cells 3:45 PM 4:00 PM James Walker, PhD, Massachusetts General Hospital

Creation of NF1-Associated Tumor Models Deficient for Polycomb Repressive Complex 2 4:00 PM 4:15 PM Kyle Williams, PhD, University of Minnesota 2:15 PM 4:15 PM PLATFORM PRESENTATIONS – CLINICAL SCIENCE

Session Chairs: Laura Klesse, MD, PhD, UT Southwestern; Martino Ruggieri, MD, PhD, University of Catania, Italy

A Multi-Center Radiomics-Based Model to Differentiate Between NF1-Associated Plexiform Neurofibromas 2:15 PM 2:30 PM and Malignant Peripheral Nerve Sheath Tumors Ina Ly, MD, Massachusetts General Hospital

Cell-Free DNA Ultra Low-Pass Whole Genome Sequencing Distinguishes Malignant Peripheral Nerve Sheath Tumor 2:30 PM 2:45 PM from its Benign Precursor Russell Sundby, MD, National Cancer Institute (NCI)

Children with NF1 Have Significant Deficits in Muscle Function 2:45 PM 3:00 PM Amish Chinoy, MBBS, University of Manchester, UK

A Randomized Placebo-Controlled Phase 2 Study of the Efficacy and Safety of Tanezumab in Subjects 3:00 PM 3:15 PM with Moderate-to-Severe Schwannomatosis Jennifer Da, BA, Massachusetts General Hospital

Long-Term Safety and Efficacy of Selumetinib in Children with Neurofibromatiosis Type 1 on a Phase 2 Study 3:15 PM 3:30 PM for Inoperable Plexiform Neurofibroma Andrea Gross, MD, National Cancer Institute (NCI)

ReNeu: Phase 2b Trial of Mirdametinib, a MEK1/2 Inhibitor in Patients with Neurofibromatosis Type 1-Associated 3:30 PM 3:45 PM Plexiform Neurofibroma Causing Significant Morbidity Christopher Moertel, MD, University of Minnesota

Binimiteinib Leads to Radiographic Response in Adults with Neurofibromatosis Type 1 Associated Plexiform 3:45 PM 4:00 PM Neurofibromas: A Report from the NFCTC and PNOC Alyssa Reddy, MD, University of California San Francisco

Predictors of Early Relapse and Treatment-Refractory Optic Pathway Glioma in Children with Neurofibromatosis Type 1 4:00 PM 4:15 PM Chelsea Kotch, MD, Children’s Hospital of Philadelphia

16 | Children’s Tumor Foundation · Ending Neurofibromatosis Through Research AGENDA Wednesday · June 16, 2021 (All times are ET)

8:30 AM 9:30 AM EUROPEAN INITIATIVES – UPDATE (NON-CE ACTIVITY)

EFPIA & IMI 8:30 AM 8:40 AM Magda Chlebus, European Federation of Pharmaceutical Industries and Associations (EFPIA)

Introduction to EU PEARL 8:40 AM 8:50 AM Francesco Patalano, MD, EU PEARL

NF Platform Trial - Template Design 8:50 AM 9:05 AM Guenter Heimann, PhD, Novartis

Platform Trial for Plexiform Neurofibroma Using the Heimann Template 9:05 AM 9:20 AM Britt Dhaenens, MD, Erasmus University

Business Model Towards Sustainability 9:20 AM 9:30 AM Edwin van de Ketterij, MSc, European Research Infrastructure for Translational Research (EATRIS) 9:30 AM 10:00 AM POSTER COMPETITION WINNERS – SHORT ORAL PRESENTATIONS 10:00 AM 11:30 AM CONSORTIA UPDATES

Department of Defense NF Clinical Trials Consortium 10:00 AM 10:20 AM Michael Fisher, MD, Children’s Hospital of Philadelphia

Optic Pathway Glioma Consortium 10:20 AM 10:40 AM Robert Avery, DO, Children’s Hospital of Philadelphia

Synodos Low Grade Glioma Consortium 10:40 AM 11:00 AM Michael Fisher, MD, Children’s Hospital of Philadelphia; David Gutmann, MD, PhD, Washington University

Perspectives on Synodos for Gliomas 11:00 AM 11:15 AM Michael Fisher, MD, Children’s Hospital of Philadelphia; David Gutmann, MD, PhD, Washington University; Antonio Iavarone, MD, Columbia University

International Schwannomatosis Database 11:15 AM 11:30 AM Allan Belzberg, MD, Johns Hopkins University

11:30 AM 11:45 AM BREAK 11:45 AM 12:15 PM LATE BREAKING ABSTRACTS – PLATFORM PRESENTATION Phase IIa Trial of Topical MEK Inhibitor, NFX-179, in Neurofibromatosis Type 1 Patients with Cutaneous Neurofibromas 11:45 AM 12:00 PM Kavita Yang Sarin, MD, PhD, Stanford University 12:15 PM 12:30 PM HACKATHON – FROM HACK FOR NF TO HACK4RARE (NON-CE ACTIVITY) Salvatore La Rosa, PhD, CSO, Children’s Tumor Foundation 12:30 PM 12:50 PM PRESENTATION OF 2021 FRIEDRICH VON RECKLINGHAUSEN AWARD 12:50 PM 1:05 PM WRAP-UP AND ADJOURNMENT 1:15 PM 5:00 PM REiNS MEETING

2021 NF Virtual Conference · June 14-16, 2021 | 17 PARTICIPANTS Attendees List – 2021 NF Virtual Conference

NAME TITLE COMPANY WORK CITY WORK COUNTRY Afridi, Shazia Dr Guy’s and St Thomas’ NHS Trust London United Kingdom Aggarwal, Anjali Assistant Professor University of Minnesota Minneapolis USA Agyei Wiafe, Samuel Mr Rare Disease Ghana Accra Ghana Akshintala, Srivandana Staff Clinician NCI-Pediatric Oncology Branch Bethesda USA Al-Naseem, Abdulrahman Manchester United Kingdom Allain, Meredith Medical Geneticist Ochsner Medical Center New Orleans USA Allaway, Robert Senior Scientist Sage Bionetworks Seattle USA Allen, Taryn Clinical Psychologist Clinical Research Directorate (CRD), Frederick National Laboratory Bethesda USA for Cancer Research Alós, Juan Carlos Rare Disease Leader AstraZeneca Madrid Spain Altman, Dan Co-Managing Partner Belkin Burden Wenig & Goldman Jericho USA Alveshere, Andrea Assistant Professor Western Illinois University Macomb USA Angulo, Ivan Pharmacology Principal Scientist Healx Cambridge United Kingdom Anstett, Kara Genetic Counselor NYU Langone Health New York USA Arslane, Samia Beverly Hills USA Askenazi, Naomi Student Massachusetts General Hospital Arlington USA Athersmith, Megan Dr NHS Manchester United Kingdom Avery, Robert Physician Children’s Hospital of Philadelphia Philadelphia USA Babovic, Dusica Clinical geneticist Mayo Clinic rochester USA Bachman, John Fellow in Therapeutic Science Harvard Medical School Boston USA Bailey, Sue Associate Director Astra Zeneca MAcclesfield United Kingdom Bakker, Annette President Children’s Tumor Foundation New York USA Balasingam, Vijayabalan Montreal Canada Baldwin, Andrea CRNP NIH Bethesda USA Banerjee, Jineta Senior Scientist Sage Bionetworks Seattle USA Barklam, Clare Sen advocate Childhood tumour trust Dudley United Kingdom Barlassina, Luigi cork Ireland Barnett Tapia, Carolina Assistant Professor University of Toronto Toronto Canada Barnett, Andy VP Global Franchise Head – Lynparza DDR, Astra Zeneca Cambridge United Kingdom Rare Cancers and Established Oncology Barut, Volkan Sr. Global Medical Affairs Leader, Head of GMA, AstraZeneca Cambridge United Kingdom Selumetinib / Rare Diseases & Oncology Beach, Paul Mr. Emden USA Beaufort-Jones, Rachel Specialist Neurofibromatosis Nurse Nerve Tumours UK London United Kingdom Bedwell, David Professor and Chair 175 Hoover USA Begbie, Natasha Dr AstraZeneca Cambridge United Kingdom Bell, Marcus NTUK Ops Manager Nerve Tumours UK London United Kingdom Belzberg, Allan Professor Johns Hopkins Baltimore USA Bénédetti, Hélène PhD CNRS Orleans France Berg, Dale Patient representative Focus Sales Minneapolis USA Bergqvist, Christina Dr. Henri Mondor Hospital Créteil France Berman, Yemima A/Prof Royal North Shore Hospital St Leonards Australia Bessler, Waylan Research Analyst Indiana University Indianapolis USA Bhunia, Minu PhD Candidate University of Minnestoa Minneapolis USA Black, Laurel PhD Candidate Medical University of South Carolina Charleston USA Blackwell, Rebecca Nurse Practitioner - Clinical Genetics Dell Children’s Medical Group - Clinical Genetics Austin USA Blakeley, Jaishri Professor of Neurology, Neurosurgery Johns Hopkins University School of Medicine Baltimore USA and Oncology Borradori, Luca Prof. Universitätsklinik für Dermatologie Bern Switzerland Boulas, Amy CEO Cupid’s Charity Denver USA Boywitt, Adriana Lic en Cs Biológicas- Citogenética Hospital de Niños R. Gutiérrez-CABA CABA Argentina Brainin and Mrs. Nicola Kean, Board Member Children’s Tumor Foundation Brookline USA Robert Branch, Jacqueline Developmental Behavioral Pediatrician Akron Children’s Hospital Akron USA Breun, Maria Dr. University Hospital Würzburg Würzburg Germany Brosseau, Jean-Philippe Assistant Professor Université de Sherbrooke Sherbrooke Canada Brossier, Nicole Instructor Washington University in St. Louis Saint Louis USA Browder, Vidya Basic Science Senior Manager Children’s Tumor Foundation New York USA Brown, Alexa Dr WAVES Perth Australia Brown, Rebecca Assistant Professor Mount Sinai Hospital New York USA Bruemmer, Kevin Postdoc Stanford University - Bertozzi Lab Stanford USA Bui, Ashley Fellow Dallas USA Buono, Frank Associate Research Scientist Yale School of Medicine Hamden USA Burckhardt, Susanna International Medical Associate Director AstraZeneca Barr Switzerland for Rare Diseases Burgess, Azura Global Development Scientist AstraZeneca Cambridge United Kingdom Burgess, Crystal Ms. National Cancer Institute Bethesda USA Burkitt Wright, Emma Dr Manchester Centre for Genomic Medicine, Manchester University NHS Manchester United Kingdom Foundation Trust Cadwgan, Jill Dr Guys’s and ST Thomas Foundation trust London United Kingdom Calizo, Ana Research Specialist Johns Hopkins University School of Medicine Baltimore USA Campen, Cynthia J. Dr Stanford School of Medicine Palo Alto USA Careels, Leen Family Doctor Solist Nijlen Belgium Carpio, Arianne SpringWorks Therapeutics Stamford USA Carrió, Meritxell Ms Germans Trias i Pujol Research Institute Badalona Spain Carton, Charlotte KU Leuven, Laboratorium voor Onderzoek van Neurofibromatose Leuven Belgium Cassels, Eoin Global Brand Director AstraZeneca Cambridge United Kingdom Castellanos, Elisabeth Ms Germans Trias i Pujol University Hospital (HUGTiP) Badalona Spain

18 | Children’s Tumor Foundation · Ending Neurofibromatosis Through Research PARTICIPANTS Attendees List – 2021 NF Virtual Conference

NAME TITLE COMPANY WORK CITY WORK COUNTRY Catasus, Nuria Ms Germans Trias i Pujol Research Institute Badalona Spain Caubo, Irene Ms UvH Dieren Netherlands Chandra, Autkarsh East Newark USA Chang, Long-Sheng Professor Nationwide Children’s Hospital and The Ohio State University Columbus USA Chang, Robert Senior Director, Field Medical SpringWorks Therapeutics Stamford USA Chi, Ping Medical Oncologist Memorial Sloan Kettering Cancer Center New York USA Chiang, Jianbang Dr National Cancer Centre Singapore Singapore Singapore Chiara, Federica Assistant Professor University of Padova Padova Italy Chistoprudova, Anna Director Interregional non-government organization for patients with neurofibromatosis “22/17” Moscow Russian Federation Chubb, Tom RDL AstraZeneca Cambridge United Kingdom Ciscato, Francesco Post-Doc University of Padua Padova Italy Clapp, David Chair, Department of Pediatrics Indiana School of Medicine Indianapolis USA Clapp, Wade M.D., Professor Department of Pediatrics, Indiana University School of Medicine Indianapolis USA Cook, Warren Mr AstraZeneca Cambridge United Kingdom Corgosinho, Marina Student CRNF Belo Horizonte Brazil Correia, Patricia Rio de Janeiro Brazil Craigen, William Professor Baylor College of Medicine Houston USA Creus, Edgar Fundació Institut d’Investigació Biomèdica de Bellvitge-IDIBELL L’Hospitalet Spain de Llobregat Croci, Carolina Child Neurologist Fondazione IRCCS Istituto Neurologico Carlo Besta Milan Italy D’Abate, Lia Post-Doctoral Fellow Hoffmann-La Roche Montreal Canada D’Agostino, Maria-Daniela MUHC Montreal Canada D’Angelo, Fulvio Postdoctoral Researcher Columbia University New York USA Da, Jennifer Clinical Research Coordinator II Massachusetts General Hospital Boston USA Dabscheck, Gabriel Dr Royal Children’s Hospital, Melbourne VIC Australia Dahbar, Myriam Buenos Aires Argentina Dahl, Hilde Margrete Pediatric Neurologist Oslo University Hospital Oslo Norway Dahlheimer, Tambra CNP University of MN Physicians. Minneapolis USA Darrigo Junior, Luiz Guilherme DR University of São Paulo Ribeirão Preto Brazil Day, Ellie Paediatrician Royal Free London NHS Trust London United Kingdom de Blank, Peter Associate Professor Cincinnati Children’s Hospital Medical Center Cincinnati USA de la Rosa Rodriguez, Randolph Medical Director AstraZeneca Clinton USA DeCillis, Stacey Albertson USA Dekker, Saskia Board Member Stichting Neurofibromatose Limmen Netherlands del Castillo, Allison Chicago USA Delgado, Kiley Genetic Counselor MGH South Hamilton USA Denayer, Ellen Dr UZ Leuven Leuven Belgium Derdak, Joanne Nurse Practitioner NIH Bethesda USA Dhaenens, Britt Drs Erasmus MC Rotterdam Netherlands Dhillon, Clare Specialist Neurofibromatosis Nurse Nerve Tumours UK London United Kingdom DiDomenico, Concetta Nurse Practitioner The Children’s Hospital of Philadelphia Philadelphia USA Dirksen, Sara Project Manager NF Denmark Vedbaek Denmark Dixit, Karan Assistant Professor Northwestern University Chicago USA Dobrea, Lavinia Clinical Science Liaison AstraZeneca Yorba Linda USA Dodd, Rebecca Assistant Professor University of Iowa Iowa City USA Dombi, Eva Associate Scientist NCI Bethesda USA Dong, Juan Lab Director UAB Birmingham USA Donnelly, Deirdre Dr Belfast Health & Social Care Trust Belfast United Kingdom Donnelly, Kelly DO Albany Medical Center Albany USA Doshi, Dipam MSL SpringWorks Therapeutics Diamond Bar USA Doughton, Gail AstraZeneca Cambridge United Kingdom Draper, Garrett PhD Student University of Minnesota Minneapolis USA Drozd, Hayley MSTP Student Indiana University School of Medicine Indianapolis USA Dubois, Anna Rare Disease Leader - France & BeNeLux AstraZeneca Courbevoie France Dufek, Anne Nurse Practitioner NIH Bethesda USA Dussán, Andrés DR Hospital Militar Central Bogotá Colombia Dye, Alison Associate Director, US Advocacy & AstraZeneca Gaithersburg USA Alliance, Oncology, Hematology/NF1 Earl, Dawn Nurse Practitioner in Genetic Medicine Seattle Children’s Seattle USA Earle, Suzanne Owner, Vice President Earle & Associates (Attending as a member of the CTF Board of Directors) St. Petersburg USA Economides, Christina Public Policy Advisor Squire Patton Boggs Brussels Belgium Eelloo, Judith Lead Nurse Complex NF1 Service Manchester University NHS Foundation Trust Manchester United Kingdom Ehrli, Hannah Special Educator New Smyrna Beach USA Ejerskov, Cecilie Postdoctoral Reseacher Center for Rare Diseases, Paediatric and Adolescent Medicine, Aarhus N Denmark Aarhus University Hospital, Aarhus Eligio, Gabriel Odontólogo Eligio & González SAS Bogotá Colombia Emmanouil, Beatrice Oxford University Hospitals NHS Trust Oxford United Kingdom Ericson, Lars Quantitative Analytics Specialist Catskills Research Company Davidson USA Espinosa, Eugenia Neurology Pediatrics 21 Invitado Bogota Colombia Esposito, Dominic Director, Protein Sciences Frederick National Laboratory Frederick USA Eubanks-Rudd, Jacquelyn Finance Manager Johns Hopkins University Baltimore USA Evans, Alison Associate Director, Oncology Patient Affairs AstraZeneca Cambridge United Kingdom Evans, D Gareth Prof UoM Manchester United Kingdom Evans, Mathew Dr Guy’s and ST Thomas NHS Foundation Trust London United Kingdom Everson, Richard Assistant Professor UCLA Los Angeles USA Ezzedine, Khaled Prof. Referral Center for Neurofibromatosis – Dermatology – Créteil France Henri Mondor Hospital – Créteil – France Farahzadi, Trish Neuro-Oncology Nurse Practitioner Stanford- Lucile Packard Childrens Hospital Palo Alto USA

2021 NF Virtual Conference · June 14-16, 2021 | 19 PARTICIPANTS Attendees List – 2021 NF Virtual Conference

NAME TITLE COMPANY WORK CITY WORK COUNTRY Farholt, Stense Consultant Center for Rare Diseases, Rigshospitalet, Copenhagen Copenhagen Denmark Farrar, Jill RN VCUHealth Richmond USA Ferk, Salah Dr Referral Center for Neurofibromatosis – Dermatology Department – Créteil France Henri Mondor Hospital – Créteil – France Fernandez-Rodriguez, Juana Ms Germans Trias i Pujol Research Institute L’Hospitalet Spain de Llobregat Fernandez-Valle, Cristina Professor University of Central Florida Orlando USA Ferner, Rosalie Professor Guy’s and St. Thomas’ NHS Foundation Trust London United Kingdom Ferrari, Silvia Rare Disease Medical Lead AstraZeneca Basiglio Italy Ferrer, Marcela María PhD Hospital de Clínicas - Universidad de Buenos Aires Ciudad Autónoma Argentina de Buenos Aires Fertitta, Laura MD Referral Center for Neurofibromatosis – Dermatology – Créteil France Henri Mondor Hospital – Créteil – France Fiesco-Roa, Moisés Associate Professor, MD, MSc UNAM Coyoacán Mexico Finamore, Lauren Nurse Practitioner Cincinnati Children’s Hospital Cincinnati USA Fisher, Michael J. Dr. Children’s Hospital of Philadelphia Philadelphia USA Flores Yucra, Giovanna Betty Neuropediatra Hospital Japonés Santa Cruz Bolivia Foreman, Nicholas Professor, Pediatrics Children’s Hospital Colorado Aurora USA Franco Bustamante, Kelly Médico Genetista Instituto Nacional de Salud del Niño San Borja Lima Peru Franklin, Barbara Patient Representative Patient Advocate Morristown USA Franzon Bonatti, Renata Cristina Teacher Universidade Federal Do Triângulo Mineirouftm Uberaba Brazil Fritz, Erin CNP University of MN Journey Clinic Minneapolis USA Frost, Michael Hematopathologist Western Diagnostic Services Laboratory Santa Maria USA Galloway, Tracy Vice Chair, Board of Directors CTF The Childrens Tumor Foundation Mooresville USA Galvin, Robert Pediatric Hematology/Oncology Fellow University of Minnesota Minneapolis USA Garcia, Natalia San Antonio USA García, Pilar Mrs GerNF Commugny Switzerland Gardner, Kathy Assistant Professor of Neurology University of Pittsburgh Pittsburgh USA Garrett, Edith Arlington USA Gel, Bernat Dr The Institute for Health Science Research Germans Trias i Pujol (IGTP) Badalona Spain Geller, Thomas Professor of Child Neurology Johns Hopkins All Children’s Hospital St Petersburg USA Gibson-Williams, Madalyn USA Gilbert, Dani Quicken Loans Detroit USA Ginsberg, Matthew Assistant Professor of Pediatrics Akron Children’s Hospital Akron USA Glissmeyer, Margaret PA-C Seattle Childrens Everett USA Goitz, Benjamin Mental Health Counselor Albany Medical Center Albany United States Minor Outlying Islands Golfinos, John NYU Langone New York USA Gomes, Alicia Genetic Counselor University of Alabama Birmingham Birmingham USA Gonzalez, Francisco International Medical Director AstraZeneca Luton United Kingdom Haematology & Rare Diseases Goss, James GTI Project Manager Gilbert Family Foundation Canonsburg USA Gottesman, Sally Board Member NTAP New York USA Goula, Maria MD Hospital Thessaloniki Greece Gourion, Anne Medical Lead AstraZeneca Rueil-Malmaison France Graber, Jerome Associate Professor University of Washington Seattle USA Granger, Leslie Genetic Counselor Randall Children’s Hospital Portland USA Greenwood, Robert Doctor UNC School of Medicine Chapel Hill USA Groisman, Gabriel Partner and Co-Chair, Trade Secrets Meland and Budwick Miami USA and IP Group Gross, Andrea Assistant Research Physician National Cancer Institute Bethesda USA Grotti, Lorenza Frenchs Forest Australia Gupta, Punita Chief, Genetics St. Joseph’s University Medical Center Paterson USA Gutmann, David Physician Washington University St. Louis St. Louis United States Minor Outlying Islands Haberkamp, Diana Executive Director Neurofibromatosis Midwest St. Charles USA Hachen, Rachel MD CHOP Philadelphia USA Hadland, Pippa Global Publications Leader AstraZenenca Cambridge United Kingdom Hagedorn, Caroline Medical Genetics Pediatric Nurse Practitioner University of Utah Salt Lake City USA Halliday, Dorothy Dr Oxford University Hospitals NHS Trust Oxford United Kingdom Halliday, Jane Oxford University Hospitals NHS Trust Oxford United Kingdom Hanemann, Clemens Oliver Professor Peninsula Medical School Plymouth United Kingdom Hanson, Amy RN Care Coordinator University of Minnesota Masonic Childrens Hospital Minneapolis USA Harder, Anja Prof. Dr. University Hospital Halle Halle Germany Hardin, Haley Graduate Student University of Central Florida Orlando USA Harrison, Rb BOD Ctf Ridgewood USA Harrod, Virginia Neuro-Oncology Dell Children’s Medical Center Austin USA Haughton, Frank Owner FH Petroleum Corp Billings USA Heiberg, Arvid Professor (em) Oslo University Hospital Oslo Norway Heimann, Guenter Franchise Head, Pharmacometrics Novartis Basel Switzerland Hemmings, Gillian Oxford University Hospitals NHS Trust Oxford United Kingdom Hendricks, Michael Resident Physician New Albany USA Henrici, Johanna AstraZeneca Wedel Germany Herfel, Kaylee RN Monona USA Hersheson, Joshua Dr Guy’s and St Thomas NHS Foundation Trust London United Kingdom Hicks, Stephanie Genetic Counselor Massachusetts General Hospital Boston USA Hirbe, Angela MD PhD Washington University School of Medicine, St. Louis St. Louis USA Hogan, Julie CPNP NCI-Pediatric Oncology Branch Bethesda USA

20 | Children’s Tumor Foundation · Ending Neurofibromatosis Through Research PARTICIPANTS Attendees List – 2021 NF Virtual Conference

NAME TITLE COMPANY WORK CITY WORK COUNTRY Horta, Erika MD University of Arkansas Medical Sciences Little Rock USA Horvitz, Richard Orange Village USA Horvitz, Richard Chairman Moreland Management Co. Orange Village USA Hou, Yang Assistant Professor University of Kentucky Lexington USA Huebsch, Jean RN Oishei Childrens Hospital of Buffalo NF Clinic Buffalo USA Huegel, Julianne Orlando USA Huerta Mercado, Jorge Dr Lima Peru Huserman, Jonathan Dr Hospital Base San José Osorno Osorno Chile Ichord, Rebecca Professor of Neurology Children’s Hospital of Philadelphia Philadelphia USA Iloeje, Uchenna Stamford USA Iltis, Charlene Research Scholar Memorial Sloan Kettering Cancer Center New York USA Inniss, Rona Mrs Guy’s and St Thomas NHS Foundation Trust London United Kingdom Irving, Carol Specialist Neurofibromatosis Nurse Nerve Tumours UK London United Kingdom Isibor, Steve Senior Medical Science Liasion Exelixis Inc. Rockaway USA Jamal, Ali Physician Saint Louis University St.Louis USA Jannic, Arnaud Dr Referral Center for Neurofibromatosis – Dermatology Department – Créteil France Henri Mondor Hospital – Créteil – France Jefferies, Samantha Occupational Therapist Harbour Farm Occupational Therapy Nottinghamshire United Kingdom Jones, Victoria Open Health London United Kingdom Jordan, Justin Assistant Professor, Clinical Director Massachusetts General Hospital Boston USA Judd, Alexis Physician Assistant Akron Children’s Hospital Akron USA (Harrison) Kalagher, Carol Ft. Lauderdale USA Kallionpää, Roope MSc (pharm) University of Turku Turku Finland Karajannis, Matthias Professor MSKCC New York USA Karwacki, Marek Ass. Professor Medical University of Warsaw Warsaw Poland Kates, Leslie Brookline USA Keeling, Kim Assistant Professor University of Alabama at Birmingham Birmingham USA Kelly-Mancuso, Geraldine NF Coordinator Cincinnati Children’s Hospital Cincinnati USA Kelts, Kate Patient Support Coordinator Children’s Tumor Foundation Champaign USA Kenyon, Tracey Specialist Neurofibromatosis Nurse Nerve Tumours UK London United Kingdom Kershner, Leah Research Fellow Cincinnati Children’s Hospital Medical Center Cincinnati USA Khan, Mohammed Azharuddin NA NA Muscat Oman Khan, Xavier Interpretation Technician Capital Linguists Silver Spring USA Khanna, Akshat Los Angeles USA Khanna, Rajesh Professor University of Arizona Tucson USA Kim, AeRang Pediatric Oncologist Childrens National Hospital Washington USA Kim, YooRi Scientific Officer Gilbert Family Foundation Detroit USA King, Lorien Clinical Coordinator St. Joseph’s Regional Medical Center Paterson USA Kirk, George Global Product Leader AstraZeneca Cambridge United Kingdom Klein-Tasman, Bonnie Professor University of Wisconsin-Milwaukee Milwaukee USA Klesse, Laura Associate Professor University of Texas Southwestern Medical Center Dallas USA Kline, Cassie Assistant Professor Children’s Hospital of Philadelphia Wynnewood USA Knight, Pamela Clinical Program Director CTF New York USA Ko, Aram Post Doctoral Research Scientist Columbia University Medical Center New York USA Koetsier, Kimberley Leiden Netherlands Kohlmeyer, Jordan Graduate Student University of Iowa Iowa City USA Korankyi, Ivana High Point USA Korf, Bruce Professor University of Alabama at Birmingham Birmingham USA Kotch, Chelsea Fellow Children’s Hospital of Philadelphia Philadelphia USA Krueger, Darcy Professor of Clinical Pediatrics Cincinnati Children’s Hospital Medical Center Cincinnati USA and Neurology Kumar, Manu Assistant Professor Maulana Azad Medical College, New Delhi New Delhi India La Serna Infantes, CEO Genocenter Lima Peru Jorge Estanislao Lage Cota, Bruno Cezar CRNF - HC/UFMG Centro de Referência em Neurofibromatoses do Hospital das Clínicas Belo Horizonte Brazil da Universidade Federal de MG Langmead, Shannon Clinic Coordinator, Adult Nurse Practitioner Johns Hopkins Comprehensive NF Center Baltimore USA Langseth, Abe Medical Director SpringWorks Therapeutics Bethesda USA Laraba, Liyam Dr Plymouth University Plymouth United Kingdom Largaespada, David Professor University of Minnesota Minneapolis USA Larocque, Viviane Medical Lead AstraZeneca Boucherville Canada Larsson, Alex Research Scientist University of Minnesota Minneapolis USA Lay-Son, Guillermo Médico, Genetista Clínico Pontificia Universidad Católica de Chile Santiago Chile Lázaro, Conxi Ms Catalan Institute of Oncology Barcelona Spain Le, Lu Professor UT Southwestern Medical School Dallas USA Lebedoff, Amy Physician Children’s Minnesota Minneapolis USA Lee, Eunice Los Angeles USA Lee, Sang Johns Hopkins University School of Medicine Baltimore USA Legault, Genevieve Pediatric Neurologist/Neuro-Oncologist McGill University Montreal Canada Lessing, Andrés REiNS Patient Representative, Milton USA NF Northeast Board Member Lester McCully, Cynthia Senior Research Associate National Cancer Institute Bethesda USA Lightner, Donita Assistant Professor University of Kentucky Lexington USA Lim, Agnes Resident Singapore General Hospital Singapore Singapore Lin, Andrew Assistant Attending Neurologist Memorial Sloan Kettering Cancer Center New York USA Lindner, Leticia Joaçaba Brazil Lingo, Joshua Iowa City USA Lipinski, Lindsay Assistant Professor of Neurosurgery Roswell Park Comprehensive Cancer Center Buffalo USA and Oncology

2021 NF Virtual Conference · June 14-16, 2021 | 21 PARTICIPANTS Attendees List – 2021 NF Virtual Conference

NAME TITLE COMPANY WORK CITY WORK COUNTRY Lipkovskaya, Vera Director for International Relations Russian Patient Association for NF “22/17” Mytishchi Russian Federation Lisbôa Basto, Diogo PhD Student Universidade Federal Fluminense Rio de Janeiro Brazil Listernick, Robert Professor of Pediatrics, Feinberg School Ann & Robert H. Lurie Children’s Hospital of Chicago Chicago USA of Medicine, Northwestern University Loewidt, Nataly Mrs NFIsrael Kfar Saba Israel Logan, Isabelle Oregon State University Corvallis USA Lou, Hua Associate Professor Case Western Reserve University Cleveland USA Lukkes, Jodi Assistant Research Professor Indiana University Indianapolis USA Lulla, Dinesh Boystown National Research Hospital Omaha USA Lunati-Rozie, Ariane Hopital Henri-Mondor Créteil France Ly, Ina Neuro-Oncologist Massachusetts General Hospital Boston USA Mackeith, Samuel Oxford University Hospitals NHS Trust Oxford United Kingdom Magallón-Lorenz, Míriam Ms Germans Trias i Pujol Research Institute Badalona Spain Maia, Rayana, Rayana MD UFPB Campina Grande Brazil Manso, Simone CTF BoD Member CTF Adliswil Switzerland Mareeva, Yulia Dr Dmitry Rogachev National Medical Research Center of Pediatric Hematology, Moscow Russian Federation Oncology and Immunology Marrs, Kimberly Graduate Student California State University, Sacramento Sacramento USA Martin Peron, Staci Dr. National Cancer Institute Rockville United States Minor Outlying Islands Martin, Linda Chairperson Emeritus Board of Directors New York USA Martin, Vanessa Chair Childhood Tumour Trust Winchelsea Beach United Kingdom Martin, Yolanda Servicio Genetica Hospital Universitario Ramon y Cajal Madrid Spain Martinelli, Maria Graduate Research Assistant University of Central Florida Orlando USA Martínez, Haydée Dr. Hospital San Martín UBA CABA Argentina Maspero, Sally Ms CTF-Australia/CHW Sydney Australia Matasconi, Manuela Miss AstraZeneca Bresso Italy Mautner, Victor-Felix Professor Department of Neurology, University Medical Center Hamburg-Eppendorf, Hamburg Germany Hamburg, Germany Mazuelas, Helena Ms Germans Trias i Pujol Research Institute Badalona Spain McClatchey, Andrea Professor MGH/Harvard Medical School Charlestown USA McCloughan, Natalie Children’s Tumour Foundation of Australia Parkville Australia McCully, Cindy Senior Research Assistant NCI-Pediatric Oncology Branch Bethesda USA McGowan, Caroline MS, CGC Boston Children’s Hospital Boston USA McKenzie, Steve Channel West Group, LLC Malibu USA McManus, Miranda College of Charleston Charleston USA McNeill, Alana Hershey USA Meade, Julia Assistant Professor of Pediatrics UPMC Children’s Hospital of Pittsburgh Pittsburgh USA Meek, Kristin Arlington Heights USA Mellado, Cecilia Clinical Geneticist/Associate Professor Pontificia Universidad Católica de Chile Santiago Chile Méndez, Gastón Santiago Chile Meneses Flores, Dr. Universidad Nacional Mayor de San Marcos Los Olivos. LIma Peru Giovanni Giuseppe Simon Merker, Vanessa L. Instructor of Neurology Massachusetts General Hospital Boston USA Merkley, Tricia RN Seattle Children’s Hospital Seattle USA Messiaen, Ludwine Professor UAB Birmingham USA Meyer, Carolyn Executive Director of Planning and Research Retired from Lake-Sumter State College Towson USA Michaels, Olivia Clinical Research Coordinator Massachusetts General Hospital Boston USA Mietchen, Jonathan Assistant Professor University of Wisconsin - Madison Madison USA Miller, David T Dr. Boston Children’s Hospital, Boston, MA, USA Boston USA Moeller, Alli Associate Director, Brand AstraZeneca Gaithersburg USA Communications - US Corporate Affairs Moertel, Chris Professor University of Minnesota Minneapolis USA Mokhtari, Sepideh Neuro-Oncologist H. Lee Moffitt Cancer Center Tampa USA Montefalcone, Giuseppe Medical Affairs Lead AstraZeneca Cambridge United Kingdom Montes, Cecilia del Carmen Jefa División Genética Medica Hospital de Niños de Cba Argentina Cordoba Argentina Morais, Pedro Scientific Director ProQR Therapeutics Leiden Netherlands Morin, Caroline Scientific Analyst Gilbert Family Foundation Detroit USA Morris, Katrina Neurologist NSW Department of Health Concord Australia Morris, Stephanie Assistant Professor Washington University Saint Louis USA Mosrin, Christine PhD CNRS Orleans France Mowatt, David Consultant Plastic Surgeon The Christie Hospital Manchester United Kingdom Moye, Jerry Physician Texas Children’s Hospital, (Retired) Frisco USA Mueller, Amy Genetic Counselor MGH Center for Cancer Risk Assessment Boston USA Mungikar, Pushkar Farmington USA Munoz, Angelica Senior Medical Science Liaison SpringWorks Therapeutics Stamford USA Murray, Bega NIH Bethesda USA Na, Brian Pediatric Hematology/Oncology Fellow UCLA Los Angeles USA Ng, Regina International Rare Diseases Brand Lead AstraZeneca Singapore Singapore Nievo, Marco PhD CTF Siena Italy Nikrad, Julia Graduate Student University of Minnesota Minneapolis USA Niño, María Eugenia CABA Argentina Nishida, Yoshihiro Professor Nagoya University Nagoya Japan Nixon, Jessica Buffalo USA Novacek, Ethan University of Minnesota Minneapolis USA Oberlander, Beverly Patient Advocate REiNS/Neurofibromatosis Network Murrieta USA Oberlander, Lewis Patient Advocate NF Network Murrieta USA

22 | Children’s Tumor Foundation · Ending Neurofibromatosis Through Research PARTICIPANTS Attendees List – 2021 NF Virtual Conference

NAME TITLE COMPANY WORK CITY WORK COUNTRY Oostenbrink, Rianne Dr ErasmusMC Sophia Rotterdam Netherlands Oribamise, Eunice Research Assistant University of Minnesota Minneapolis USA Ortega, Sara Fundació Institut d’Investigació Biomèdica de Bellvitge-IDIBELL L’Hospitalet Spain de Llobregat Ortiz, Díana Admón Hospitalaria Colmedica Bogotá Colombia Ostrow, Kimberly L. Assistant Professor of Neurology Johns Hopkins University Baltimore USA Osum, Sara Post-Doctoral Researcher University of Minnesota Minneapolis USA Pacot, Laurence Dr Cochin Institute Paris France Pancza, Patrice VP External Relations CTF New York USA Panzer, Karin Genetic Counselor - Clinic Coordinator University of Iowa Hospitals and Clinics Iowa City USA Pardej, Sara Graduate Student University of Wisconsin-Milwaukee Milwaukee USA Park, SuJung Assistant Research Professor Indiana University Indianapolis USA Parkinson, David Professor of Neuroscience University of Plymouth Plymouth United Kingdom Parma, Diana Lidia Bioquímica, Farmacéutica Universidad de Buenos Aires Buenos Aires Argentina Parra Checa, Alba Neuropsychologist Sant Joan de Deu Hospital Parry, Allyson Oxford University Hospitals NHS Trust Oxford United Kingdom Patel, Amish Memorial Sloan Kettering Cancer Center New York USA Patel, Neha cleveland USA Patel, Parth University of Central Florida College of Medicine Orlando USA Patrascu, Elena Bucharest Romania Patritti Cram, Jennifer PhD Candidate Cincinnati Children’s Hospital Cincinnati USA Patterson, Anne Director of Patient and Clinic Outreach Neurofibromatosis Northeast East Aurora USA Peltonen, Juha Dr. University of Turku Turku Finland Peltonen, Sirkku Professor University of Gothenburg Göteborg Sweden Pemov, Alexander Dr. NCI Rockville USA Peña Segura, Maria Loreto Dra. Santiago Chile Perez-Becerril, Cristina Research Associate The University of Manchester Manchester United Kingdom Perfetti, Laura Glen Cove USA Perrino, Melissa Instructor Cincinnati Children’s Hospital Medical Center Cincinnati USA Peterson, Michael D2D Productions Pacific Palisades USA Pianovski, Mara Albonei Medical Doctor Liga Paranaense de Combate ao Câncer Curitiba Brazil Piccoli, Giuseppe Senior Medical Director AstraZeneca Scarsdale USA Piccolo, Gianluca Dr. Giannina Gaslini Institute - University of Genova Genova Italy Piotrowski, Anna MD MSKCC New York USA Plotkin, Scott Giovanni Armenise - Harvard Professor Massachusetts General Hospital Boston USA of Neurology Plunkett, Caitriona Manchester Metropolitan University Manchester United Kingdom Pollard, Kai Senior Research Specialist Johns Hopkins University Pond, Dinel Genetic Counselor Children’s Minnesota Minneapolis USA Popescu, Flavia University of Minnesota Minneapolis USA Post, Leonard CSO Vivace San Mateo USA Powers, Kelly Pediatric Nurse Practitioner Children’s Hospital Los Angeles Los Angeles USA Powlovich, Lauren Associate Chief Medical Officer Focused Ultrasound Foundation Charlottesvle USA Pratilas, Christine A Associate Professor of Oncology Johns Hopkins University School of Medicine Baltimore USA Puccetti, Diane Associate Professor of Pediatrics University of Wisconsin School of Medicine and Public Health Madison USA Quelle, Dawn Professor University of Iowa Iowa City USA Radtke, Heather Ms. Children’s Tumor Foundation; Medical College of Wisconsin New York USA Rai, Puneet Genetic Counselor Oregon Health & Science University Portland USA Ramesh, Vijaya Professor Massachusetts General Hospital Boston USA Ranger, Adrianna Associate Professor Clinical Neurological London Health Sciences Centre, Schulich School of Medicine and Dentistry, London Canada Sciences-Neurosurgery Western University Ratner, Nancy Professor Cincinnati Children’s Hospital Medical Center Cincinnati USA Raymond, Staci Deerfield USA Reardon, David Clinical Director, Center for Dana Farber Cancer Institute Boston USA Neuro-Oncology Redal, Maria Ana Bióloga Molecular, Especialista en Division Genetica. Hospital de Clinicas Jose de San Martin. Univ de Buenos Aires Argentina Genética, PhD Buenos Aires Reddy, Alyssa Director of UCSF Neurofibromatosis Program UCSF San Francisco USA Redman, Carolyn Oxford University Hospitals NHS Trust Oxford United Kingdom Rednam, Surya Assistant Professor Baylor College of Medicine Houston USA Reese, Mark COO YDesign Group Walnut Creek USA Reilly, Karlyne Director, Rare Tumor Initiative NCI Bethesda USA Rennie, Andrea NF Nurse Navigator Children’s Hospital of Philadelphia Philadelphia USA Rennison, Rebecca Specialist Neurofibromatosis Nurse Nerve Tumours UK London United Kingdom Repetto, Gabriela Genetista Clinica Clinica Alemana Universidad del Desarrollo Santiago Chile Reyes, Carlos Dr. Heredity Quito Ecuador Reynolds, Kelsey Paterson USA Rezende, Nilton Coordinator, Attending Physician CRNF Minas Gerais Brazil Rhodes, Steven Fellow, Pediatric Hematology-Oncology Indiana University School of Medicine/Riley Hospital for Children Indianapolis USA Riccardi, Vincent Director The Neurofibromatosis Institute La Crescenta USA Ritchie, Karleen Oncology Clinical Science Liaison AstraZeneca Chicago USA Rivera, Barbara Dr IDIBELL, Hospitalet de Llobregat Barcelona Spain Roberts, Doug Shrewsbury USA Roberts, Joshua Baltimore USA Robinson, J. Elliott Assistant Professor Cincinnati Children’s Hospital Medical Center Cincinnati USA Robles Lopez, Karla Research Associate, NF Clinic Coordinator University of Texas at Austin, Dell Medical School Austin USA Rodbell, Liz Steve Maddden New York USA

2021 NF Virtual Conference · June 14-16, 2021 | 23 PARTICIPANTS Attendees List – 2021 NF Virtual Conference

NAME TITLE COMPANY WORK CITY WORK COUNTRY Rodrigues, Luiz Coordinator, Attending Physician CRNF Belo Horizonte Brazil Rodrigues, Luiza Professor Faculdade de Ciências Médicas de MG Belo Horizonte Brazil Rodriguez Clavijo, Blanca Doris Neurology Child Rehabilitación Regional Center of Boyacá Duitama Colombia Rodriguez, Fausto Baltimore USA Romo, Carlos Assistant Professor Johns Hopkins University Baltimore USA Rosser, Tena Interim Division Chief Children’s Hospital of Los Angeles Los Angeles USA Rubenstein, Allan Clinical Professor Comprehensive Neurology Services, PC New York USA Rudd, Ken Board Member CTF New York USA Rukin Gold, Deborah Pediatric Neurologist Rainbow Babies and Children’s Hospital Cleveland USA Rustad, Cecilie Consultant Clinical Geneticist Oslo Norway Sablina, Anna Associate Professor VIB KU Leuven Center for Cancer Biology Leuven Belgium Sack, Kelli Huntington Beach USA Sala, Louell Brooklyn USA Salazar, Ruby UCSF San Francisco USA Sale, Matthew Postdoc UCSF San Francisco USA Saletti, Veronica Pediatric Neurologist Fondazione IRCCS Istituto Carlo Besta Milan Italy Sallam, Abdelrahman Medical Student University of Manchester Manchester United Kingdom Salvador, Hector Pediatric Oncologist Sant Joan de Déu Hospital Esplugues de Spain Llobregat Sammons, Josh Scientist 1 University of Alabama at Birmingham Birmingham USA Sanders, Gregory Fellow CSIS Silver Spring USA Santoro, Claudia Dr Vanvitelli University of Campania Naples Italy Sargen, Michael Clinical Fellow National Cancer Institute Rockville USA Sarnoff, Herb Founder/CEO Infixion Bioscience, Inc. Escondido USA Scantamburlo, Francesca Padua Italy Schorry, Elizabeth Professor of Clinical Pediatrics, Emeritus Cincinnati Children’s Hospital Cincinnati USA Schuricht, Kristina Hamburg Germany Schutt, Samantha ARNP University of Iowa Iowa City USA Serra, Eduard Reseracher IGTP Badalona Spain Setlow, Carolyn Washigton Depot USA Shah, Nidhi Attending Physician Penn State Health Hershey USA Shah, Nidhi Attending physician Penn State Health Hershey USA Shaver, Kristen Austin USA Sheehy, Mackenzie Minneapolis USA Sheerin, Una Guy’s and St Thomas’ NHS Foundation Trust London United Kingdom Sherman, Larry Professor Oregon Health & Science Univsersity Beaverton USA Sidhu, Alpa Clinical Associate Professor University of Iowa Hospitals and Clinics Iowa City USA Siegel, Benjamin Child Neurology Resident Emory University School of Medicine Atlanta USA Silber, Sara Bethesda USA Silva Corgosinho, Elisa Med Student Faculdade de Ciências Médicas de Minas Gerais Belo Horizonte Brazil Silver, Amanda Pacific Palisades USA Silvestre, John Médico Neuropediatra Hospital Universiario San Jorge de Pereira Pereira Colombia Simpson, Brittany Clinical Geneticist / Assistant Professor Cincinnati Children’s Hospital Medical Center Cincinnati USA Singhal, Khushboo Delhi India Sledd, Makenzie Physical Therapist St. Louis Children’s Hospital O’Fallon USA Slobogean, Bronwyn Physician Assistant Johns Hopkins University Baltimore USA Smith, Abbi Research Analyst Indiana University School of Medicine Indianapolis USA Smith, Jan New York USA Smith, Katherine NF Clinical Research Nurse Children’s Hospital of Philadelphia Philadelphia USA Smith, Miriam Senior Lecturer in Cancer Genomics University of Manchester Manchester United Kingdom Smyth, Carolyn Specialist Neurofibromatosis Nurse Nerve Tumours UK London United Kingdom Snajczuk, Jordan Genetic Counselor VCU Health Richmond USA Soll, Richard Senior Adviser, Strategic Initiatives WuXi AppTec Cambridge USA Sorge, Eberhard Universitätsklinikum Halle (Saale), Institut für Pathologie Halle (Saale) Germany Souza, Juliana Ms. Federal University of Minas Gerais Belo Horizonte Brazil Stanicky, Randall Managing Director RBC Capital Markets New York USA Stavinoha, Pete Professor University of Texas MD Anderson Cancer Center Houston USA Stemmer, Anat Associate Professor, Neuropathologist Massachusetts General Hospital Boston USA Stern, Edward Attorney Children’s Tumor Foundation Waban USA Stevenson, David Professor Stanford University Stanford USA Storozhuk, Olha Justus-Liebig University of Giessen Giessen Germany Struemph, Kari Kansas City USA Sun, Peng Sr. Global Development Medical Director AstraZeneca Gaithersburg USA Suna, Stuart Board of Directors Long Island CIty USA Sundby, Taylor Pediatric Hematology-Oncology Fellow National Cancer Institute- Pediatric Oncology Branch Rockville USA Sverak, Pavlina Staff University of Minnesota Minneapolis USA Swann, Helen Specialist Neurofibromatosis Nurse Nerve Tumours UK London United Kingdom Tang, Tracy Dr Vivace Therapeutics San Mateo USA Tang, Vivian Consultant Paediatric Radiologist Royal Manchester Children’s Hospital Manchester United Kingdom Taylor, Ashley Physician Assistant OU Health OKC USA Taylor, Ros Southmead Hospital Bristol United Kingdom Teitelbaum, Jennifer AstraZeneca Canada Mississauga Canada Téllez Zambrano, Juanita Bogotá Colombia Thomas, Elaine Bethesda USA Thompson, Andrew Open Health Marlow United Kingdom Thompson, Heather Associate Professor California State University Sacramento Sacramento USA

24 | Children’s Tumor Foundation · Ending Neurofibromatosis Through Research PARTICIPANTS Attendees List – 2021 NF Virtual Conference

NAME TITLE COMPANY WORK CITY WORK COUNTRY Tibery, Cecilia Physician Assistant Leidos/POB at NIH Bethesda USA Tödter, Carola Rare Disease Lead DACH AstraZeneca Wedel Germany Toledano Alhadef, Hagit Dr Gilbert Israeli and International Center, Tel Aviv Sourasky Medical Center Tel Aviv Israel Tomkins, Helen Oxford University Hospitals NHS Trust Oxford United Kingdom Torres Nieto, Maria Paola Genetista Consultorio Particular Valledupar Colombia Tovar Ayala, Mar Gabriela Médico Especialista en Genética Médica Life in Genomics México city Mexico Trevisson, Eva Prof University of Padova Padova Italy Truitt, Morgan Postdoctoral Fellow The Salk Institute for Biological Studies La Jolla USA Ullrich, Nicole Associate Professor Boston Children’s Hospital Boston USA Uriarte-Arrazola, Itziar Ms Germans Trias i Pujol Research Institute Badalona Spain Valinotto, Laura Investigator R Gutiérrez Children’s Hospital Buenos Aires Argentina Vallee, Beatrice PhD CBM-CNRS ORLEANS France van Dam, Victor MD Leiden University Medical Center Leiden Netherlands van Dijk, Sarah MSc Erasmus MC Rotterdam Netherlands Vaseva, Angelina Assistant Professor University Of Texas Hlth Sci Ctr San Antonio, Greehey Children’s Cancer San Antonio USA Research Institute Vassallo, Grace Consultant Paediatric Neurologist Manchester University Hospitals NHS Foundation Trust, Manchester, UK Manchester United Kingdom Vater, Mary Rose Oncology Nurse Coordinator Roswell Park Comprehensive Cancer Center Buffalo USA Velez-van-Meerbeke, Alberto Médico Neuropediatra Universidad del Rosario Bogota Colombia Ventola, Michelle Program Coordinator Kaleida Health Buffalo USA Villaprado Santana, Jose Miguel Psiquiatra HGSD Santo Domingo Ecuador Vinnakota, Kalyan Scientific Project Manager Gilbert Family Foundation Detroit USA Vishwanath, Vijay Assistant Professor, Pediatrics and Neurology Children’s Hospital Los Angeles Los Angeles USA Viskochil, David Professor University of Utah Salt Lake City USA Vitte, Jeremie Associate Project Scientist UCLA Los Angeles USA Vogels, Annick MD PhD University Hospital of Leuven Leuven Belgium Walbert, Tobias Director NF Clinic Henry Ford Health System Detroit USA Walker, Nate Investors Mgmt Co, Inc. Tarzana USA Wallace, Peggy Professor Univ. of Floridia Gainesville USA Wallis, Deeann Associate Professor UAB Birmingham USA Walsh, Karin S. Neuropsychologist, Associate Professor Children’s National Hospital/George Washington University SOM Catonsville USA Wang, Jiawan Research Associate Johns Hopkins University School of Medicine Baltimore USA Wang, Xia Medical Geneticist Moffitt Cancer Center Tampa USA Wang, Zhihong Dr. Children’s Hospital of Richmond at Virginia Commonwealth University, Richmond USA Richmond, VA, USA Watson, Adrienne Vice President of Research and Development Recombinetics Eagan USA Weber, Michael Associate Director, Clinical Development SpringWorks Therapeutics Durham USA Weintraub, Lauren Associate Professor Albany Medical Center Albany USA Weisman, Allison Genetic Counselor Ann & Robert H. Lurie Children’s Hospital of Chicago Chicago USA Weiss, Brian Professor Of Clinical Pediatrics Cincinnati Children’s Hospital Cincinnati USA Whitcomb, Trish Clinical Manager NCI-Pediatric Oncology Branch Bethesda USA Wickiser, Jonathan Dallas USA Widermann, Brigitte Branch Chief NCI-Pediatric Oncology Branch Bethesda USA Williams, Kyle Postdoctoral Fellow Masonic Cancer Center, University of Minnesota Minneapolis USA Wilpon, Clara New York USA Wimmer, Sarah RN Children’s Minnesota Minneapolis USA Winer, Rachel Dr. Seattle USA Wojtkiewicz, Agnieszka PhD Warsaw Poland Wolkenstein, Pierre Prof. Referral Center for Neurofibromatosis – Dermatology – Créteil France Henri Mondor Hospital – Créteil – France Wolters, Pamela Director, Health Psychology and Neurobehavioral National Cancer Institute Bethesda USA Research; Co-Director, Behavioral Health Core Woodall, Lesli Assistant Professor, Assistant Program Director Medical University of South Carolina Charleston USA Woolrich, Rachel Oxford University Hospitals NHS Trust Oxford United Kingdom Wright, Erin MD Akron Children’s Hospital Akron USA Wu, Jianqiang Associate Professor Cincinnati Children’s Hospital Cincinnati USA Wu, Natalie Research Associate CCHMC Cincinnati USA Xixis, Kathryn Child Neurologist University of Virginia Charlottesville USA Yarosky, Ronit Project Manager KBF CANADA Montreal Canada Ye, Manuela Paris France Yohay, Kaleb Associate Professor Comprehensive Neurofibromatosis Center at NYU Langone Health New York USA Yoshida, Yuichi Associate Professor Tottori University Hospital Yonago Japan Yoshinaga, Akiko Doctor Kyoto University Hospital Brookline USA Young, Lucy Dr UCSF San Francisco USA Yskout, Koen Parent Tongeren Belgium Yüce Petronczki, Özlem Boehr Wien Austria Zalmanek, Brynn Bioinformatics Analyst, Data Curation Sage Bionetworks Seattle USA Zambrano Vera, Dario Fernando Especialista en Neurologia PCT Manta Ecuador Zampelli, Annette CSL AstraZeneca Marysville USA Zehou, Ouidad MD Referral Center for Neurofibromatosis – Dermatology Department – Créteil France Henri Mondor Hospital – Créteil – France Zhang, Lindy Clinical and Research Fellow Johns Hopkins Baltimore USA Zhang, Xiyuan Research Fellow National Cancer Institute Bethesda USA Zheng, Bin Associate Professor MGH, Harvard Med School Boston USA Zhou, Jiangbing Associate Professor Yale University New Haven USA Zondani, Zizipho London United Kingdom

2021 NF Virtual Conference · June 14-16, 2021 | 25 OTHER MEETINGS AND PROGRAMS

26 | Children’s Tumor Foundation · Ending Neurofibromatosis Through Research 2021 NF Virtual Conference · June 14-16, 2021 | 27 Faculty Judge Mentors

Call for Abstracts Open Deadline: June 23rd at 11:59 PM ET Submit here: www.ceconcepts.com/nfyif

Who Should Submit? • North American-based Junior Faculty, Fellows, and Postdoctoral Researchers (MDs, DOs, and PhDs) involved in neurofibromatosis research. • Junior Faculty (Instructors or Assistant Professors not more than 5 years from first

• faculty• appointment) • Clinician Scientist (MD/DO +/- PhD in active patient care and conducting any lab- based or clinical research in neurofibromatosis) • Research Scientist (MD/DO and/or PhD conducting full time lab-based research in neurofibromatosis) • Fellows and Postdoctoral Researchers (Must be enrolled in an ACGME-accredited fellowship program or have completed a PhD program within the past 5 years) • • • Clinical Fellow (MD/DO +/- PhD conducting any clinical or lab-based research in

• neurofibromatosis)• • PhD Postdoctoral Researcher (PhD conducting lab-based research in neurofibromatosis)

28 | Children’s Tumor Foundation · Ending Neurofibromatosis Through Research 2021 NF Virtual Conference · June 14-16, 2021 | 29 30 | Children’s Tumor Foundation · Ending Neurofibromatosis Through Research 2021 NF Virtual Conference · June 14-16, 2021 | 31 SPECIAL THANKS TO THE 2021–2022 NF CONFERENCE CO-CHAIRS

Thomas De Raedt, PhD, Children’s Hospital of Philadelphia

Laura Papi, MD, University of Florence, Italy

AND THE 2020 NF CONFERENCE CO-CHAIRS

Matthias Karajannis, MD, MS, Memorial Sloan Kettering Cancer Center

Conxi Lázaro, PhD, Catalan Institute of Oncology

Nicole J Ullrich, MD, PhD, Boston Children’s Hospital

OUR THANKS ALSO TO THE FOLLOWING INDIVIDUALS FOR THEIR EFFORTS:

Scientific Review Committee Members

Annette Bakker Mathias Karajannis Jonathan Payne Miriam Bornhorst Rajesh Khanna Sirkku Peltonen Elisabeth Castellanos AeRang Kim Heather Radtke Ping Chi Laura Klesse Steven Rhodes Thomas DeRaedt Pamela Knight Vincent Riccardi Rebecca Dodd Salvatore La Rosa Tena Rosser Eva Dombi Conxi Lazaro Larry Sherman Rosalie Ferner Lu Le Miriam Smith Marco Giovannini Roberet Listernick Juliana Souza David Gutmann Staci Martin Piotr Topilko Angela Hirbe Christopher Moertel Karin Walsh Antonio Iavarone Marco Nievo Brian Weiss Justin Jordan Rianne Oostenbrink Pamela Walters Michel Kalamarides Laura Papi Kaleb Yohay Eric Pasmant

32 | Children’s Tumor Foundation · Ending Neurofibromatosis Through Research SPONSORS Sponsors and Supporters

CTF would like to thank our sponsors and supporters:

2021 NF Virtual Conference · June 14-16, 2021 | 33 34 | Children’s Tumor Foundation · Ending Neurofibromatosis Through Research 2021 NF Virtual Conference · June 14-16, 2021 | 35