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2020 VIRTUAL NF CONFERENCE JUNE 15 - 16, 2020 Keeping the NF Community Connected

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2020 VIRTUAL NF CONFERENCE JUNE 15 - 16, 2020 Keeping the NF Community Connected 2020 VIRTUAL NF CONFERENCE JUNE 15 - 16, 2020 Keeping the NF community connected CONTENTS Table of Contents INFORMATION Welcome Message .................................................................................................................4 Foundation Staff .....................................................................................................................5 Advisory Boards ....................................................................................................................6 Funding Opportunities ............................................................................................................7 The Friedrich von Recklinghausen Award: NF Tradition and Progress .......................................8 Schedule at a Glance ..............................................................................................................9 Conference Co-Chairs ..........................................................................................................10 Keynote Speaker ..................................................................................................................11 Agenda ................................................................................................................................12 Acknowledgments ................................................................................................................72 Sponsor ...............................................................................................................................73 Participants ..........................................................................................................................74 SPEAKER ABSTRACTS Abstracts .............................................................................................................................14 POSTER ABSTRACTS NF1: Basic Science ..............................................................................................................26 NF1: Clinical Science ............................................................................................................41 NF2: Basic Science ..............................................................................................................64 NF2: Clinical Science ............................................................................................................68 2020 NF Virtual Conference · June 15-16, 2020 | 3 Dear NF Conference Attendees, I am thrilled to welcome you to the 2020 virtual NF Conference on behalf of the Children’s Tumor Foundation. For most of the world, 2020 is mainly only a sad year characterized by isolation, separation, and disease. However, for the NF community, this year already has a few major first-ever wins: the first-ever drug approval for NF1, the first-ever platform trial for NF2, and the first-ever clinical trials for schwannomatosis, and now the first-ever virtual NF Conference. For CTF and the NF community, I hope 2020 will be remembered as a year of innovation, creativity, and the strength of a community that is not disrupted by a virus, but keeps standing united to end NF. It is, therefore, our absolute honor to bring the work of our ever-strong community together virtually. Even if COVID has tried to stop us from coming together, this year’s amazing co-chairs, Drs. Matthias Karajannis, Conxi Lazaro, and Nicole Ullrich, collaborated to create a shortened but powerful virtual event. Since we all hope that we will be able to be united in person at the NF conference in Rotterdam in December, for this meeting, we have decided to bring the ‘hottest’ NF advancements to your living room! We want to make sure that all the new exciting discoveries are shared so that the field is not slowed down by this virus. The agenda is shorter than usual, but it is maximally inclusive to reach all the time zones. It will be a truly global event. We will celebrate the 30th anniversary of the discovery of the NF1 gene with no one less than Dr. Francis Collins, who is the only keynote and opening speaker this year. He has taken the time to make an amazing opening video for you. This year’s conference will be one track only. Following the clinical care symposium, all 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 are still ample opportunities to interact. We will also take a moment to celebrate: the 30th anniversary of the NF1 gene discovery and the Koselugo approval. I wish you a short but still enriching, inspiring, and fun days. Enjoy and be inspired by great successes and discoveries. While we all hope to be together again soon, in the meantime, let’s never stop our efforts to end NF! 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 Research and Medical Programs Executive Members Salvatore La Rosa, PhD - Chief Scientific Officer Richard Horvitz - Chair Elizabeth Oliver - Senior Executive Assistant Tracy Galloway - Vice Chair Vidya Browder, PhD - Basic Science Senior Manager Randall Stanicky - Treasurer Kate Kelts - Patient Support Coordinator Gabriel Groisman - Secretary Pamela Knight - Director, Clinical Program Patrice Pancza - Vice President, External Relations Board Members Heather Radtke - NF Clinic Network Manager Daniel Altman Traceann Rose - Director, Patient and Volunteer Engagement Amy Boulas Dani Williams - External Relations Coordinator Robert Brainin Daniel Gilbert Development John Golfinos Michele Przypyszny - Chief Advancement Officer Linda Halliday Martin Claudia Becerra - Manager, Walk Carol Harrison Kalagher RB Harrison Allison Cote - Senior Manager, Donor Relations Bruce Korf Emily Crabtree - Director, Development Operations Simone Manso Allyson Douglass - Development Manager, Special Events Steven L. McKenzie Angela Dumadag - Senior Manager, Endurance Renie Moss Barbara Gallagher - Vice President, Development Laura Perfetti Julie Pantoliano - Senior Manager, Youth Programs Michael Peterson Kim Robinson - Development Manager, Special Events Scott Plotkin Connie Sorman - Senior Manager, Stewardship & Volunteer Development Liz Rodbell Rebecca Taylor - Senior Manager, Walk Kenneth Rudd Kelly Vigil - Endurance Coordinator Richard Soll Jennifer White - Development Manager, Walk Stuart Match Suna Finance and Administration Advisory Board Members Sarah Bourne - Vice President, Finance 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 Renie Moss Marketing and Communications Simon Vukelj - Chief Marketing Officer Honorary Directors Albert Diaz - Director, IT and Salesforce Administration Suzanne Earle Rebecca Harris - Public Relations Senior Manager Caroyln E. Setlow Alissa Marks - Marketing Senior Manager Nate Walker Susanne Preinfalk - Design Director Ed Stern - Pro Bono Counsel Vanessa Younger - Communications Director Allan Rubenstein, MD - Director of Medical Affairs Emeritus 2020 NF Virtual Conference · June 15-16, 2020 | 5 ADVISORY BOARDS Children’s Tumor Foundation Advisory Boards Clinical Care Advisory Board Business Advisory Council Scott Plotkin, MD (Chair), Massachusetts General Hospital Randall Stanicky (Co-Chair) Oliver Adunka, MD, Ohio State University Alex Zisson (Co-Chair) Jaishri Blakeley, MD, Johns Hopkins University Lee Babiss, PhD Reema Bogin - NF2 Patient Representative Anna Barker, PhD Gary Brenner, MD, PhD, Massachusetts General Hospital Rob Brainin Alwyn Dias MSW, ACC - NF1 Paitient Representative Steven Friend, PhD Jan Friedman, MD, PhD, University of British Columbia Stephen Groft, PharmD Michele Holbrook - Schwannomatosis Patient Representative Edward Stern Jennifer Janusz, PsyD, ABPP-Cn, Children’s Hospital Colorado Vicki Match Suna, AIA Justin Jordan, MD, MPH, Massachusetts General Hospital Bonnie Klein-Tasman, PhD, University of Wisconsin Milwaukee Research Advisory Board Laura Klesse, MD, PhD, UT Southwestern Medical Center D Wade Clapp, MD, PhD (Chair), Indiana University Pamela Knight, MS - CTF Staff Nader Fotouhi (DDIRR) - member Heather Radtke, MS, CGC- CTF Staff Jennifer Janusz (CRA) - member Tena Rosser, MD, Children’s Hospital of Los Angeles Bonnie Klein-Tasman (CRA) - member Elizabeth Schorry, MD, Cincinnati Children’s Hospital Andrea McClatchey (YIA) - member Pamela Trapane, MD, UF Health Jacksonville Tena Rosser (CRA) - member Nicky Ullrich, MD, PhD, Boston Children’s Hospital Nicky Ullrich (CRA) - member David Viskochil, MD, PhD (Chair, Emeritus), University of Utah Georg Terstappen (DDIRR) - member Kaleb Yohay, MD, NYU Langone Medical Center Peggy Wallace (YIA) (Chair Emeritus) - member Members of the RAB are also the 50+ experts and patient Medical Advisory Board representatives that agreed to conduct rigorous peer-review Bruce Korf, MD, PhD (Chair), University of Alabama at Birmingham of scientific proposals Salvatore La Rosa, PhD, CTF Chief Scientific Officer Annette Bakker, PhD, CTF President Jaishri Blakeley, MD, Johns Hopkins University Wade Clapp, MD, Indiana University Suzanne Earle, CTF Member of BOD Michael Fisher,
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