Year in Review 2019

Dear Friends of HDSA, Are we where we want to be? NOT YET. Are we getting closer? YES! With more hands on deck, we are able Putting all the great advancements in research aside, it is important to improve our tremendous support to be reminded that there are still thousands of families impacted programs even more, such as by Huntington’s disease that are in dire need of support right now. HDSA’s telehealth program, online Although we are excited to report on promising research news — support groups, HD Trialfinder and TABLE OF CONTENTS there is still no treatment for HD and our work is not done. In 2019 helpline — All free. All the time. we worked hard to expand these services to provide world-class HDSA is on the forefront of PRESIDENT & CEO MESSAGE 1 support to families from coast to coast. supporting promising HD research. This year we expanded our Centers of Excellence program to 47 In 2019, we funded more HD science 2019 HIGHLIGHTS 2 facilities and 6 clinical sites across America where more families than ever before including more than can receive expert care. With new Centers in California, Illinois, $1.4 million in direct grants to scientists. We also 2019 REGIONAL EVENT HIGHLIGHTS 14 New York and North Dakota, HDSA now offers care locations in 31 continued to cultivate strong relationships with leading RESEARCH FUNDING PROGRAMS 16 States plus the District of Columbia. This year, HDSA awarded a total bio-pharmaceutical partners. of $1.4 million to the Centers of Excellence program, an almost eleven As our founder Marjorie Guthrie said, “We must continue to 2019 MISSION REPORTS 20 percent increase in funding from 2018. search for HD families everywhere.” Through strategic outreach and Research 22 The Huntington’s Disease Society of America also added new staff engagement efforts, HDSA has generated more awareness than ever Advocacy 23 positions to play integral roles in supporting HD families. In June, before. We are getting more influencers involved who are using their Education 24 we welcomed Allison Bartlett, Esq. to serve as HDSA’s Manager of platforms to bring HD families together and let them know how to Disability Programs. Allison has been able to assist countless families find the help they need. Youth Programs Report 25 through the complex disability process as well as host Disability Chats Support & Services Report 26 Once again, our work is not done, but because of you we are able to provide valuable insight into social security and what is needed for to let HD know — we are never giving up this fight until we are in Disability Programs Report 27 approval. a world free of Huntington’s disease. 35TH ANNUAL HDSA CONVENTION 28 We know youth are a powerful voice in the fight against HD. Young adults and children from HD families continue to be wonderful Sincerely, MISSION & VISION STATEMENTS Inside Back Cover advocates and their passion is never overlooked. However, we knew we needed to create more support programs for youth impacted by BOARD OF TRUSTEES Inside Back Cover HD which is why we welcomed, not one, but two youth social workers to the HDSA team. Thanks to the Griffin Foundation, Katie Dykman, STAFF LEADERSHIP Inside Back Cover LISW and Jessica Marsolek, LGSW are professionals trained to serve Louise Vetter young people and respond to the unique needs of children, teens and President & Chief Executive Officer young adults impacted by HD.

1 2019 Highlights

HDSA CENTERS OF EXCELLENCE GROW TO 47 SITES ACROSS THE U.S. HDSA AWARDS INAUGURAL CLINICAL RESEARCH PILOT GRANTS TO FOUR CENTERS OF EXCELLENCE In 2019 HDSA announced that forty-seven out- In December, HDSA awarded four grants under an opportunity for HDSA Center of Excellence Applications for this grant mechanism were open standing Huntington’s disease care facilities with a new research initiative, the HDSA Center of directors and staff to get a new research ideas to all 47 HDSA Centers of Excellence and their six six satellite sites were awarded the designation of Excellence Clinical Research Pilot Program. off the ground. partner sites. Ultimately, grants were awarded to HDSA Centers of Excellence. These grants, totaling $50,000, represent HDSA’s four research teams on the following projects: “I am excited about this year’s recipients because patient-centric research focus by fostering novel This was an increase from 43 in 2018, and from just their innovative proposals could positively impact HDSA Center of Excellence Partner Site at Oregon clinical research projects within the HDSA Center 20 in 2015. The four new 2019 Centers of Excellence the way HD care and counseling are delivered, how Health & Science University, Dr. Amie Hiller of Excellence network. are: OSF HealthCare Illinois Neurological Institute symptoms are treated and how disease progression Measuring Cortisol Levels in Persons (Peoria IL), Sanford Health, (Fargo, North Dakota), These pilot projects will unite scientists with is monitored,” said George Yohrling, PhD, Chief with Huntington’s Disease University of Buffalo (New York) and University of patients through the direct participation of HD Scientific Officer and Chief Mission Officer HDSA Center of Excellence at Rush University California, Irvine. The HDSA Centers of Excellence OSF HealthCare Illinois Sanford Health, families in clinical research. The funding provides at HDSA. Medical Center, Dr. Deborah Hall are multi-disciplinary care teams with expertise in Neurological Institute (Peoria, Illinois) (Fargo, North Dakota) Optimization of Tele-Genetic Counseling Huntington’s disease that share an exemplary for Huntington’s Disease commitment to bringing comprehensive care. HDSA Center of Excellence at University of Texas The strategic expansion of the Center of Excellence Health Science Center, Dr. Erin Furr-Stimming program allows HDSA to increase access to expert Melatonin for HD Gene Carriers with HD Related HD clinical care and clinical trial opportunities to Sleep Disturbance more families across the United States. With new Centers in California, Illinois, New York and HDSA Center of Excellence at the University North Dakota, HDSA now offers care locations of Virginia, Dr. Madaline Harrison in 31 states plus the District Training in Primary Palliative Care for Huntington’s Disease of Columbia. This year, HDCARE Clinic, Gottschalk Medical Plaza, UBMD Neurology, “Our HDSA Centers of Excellence are at the HDSA awarded a total of University of California, Irvine University of Buffalo (New York) epicenter in the search for better HD care and $1,418,684 to the Centers treatments. HDSA is pleased to support these pilot of Excellence program, “HDSA is deeply committed to helping families team approach to Huntington’s disease care and programs in the confidence that our investment an almost eleven percent affected by Huntington’s disease have access to research. Patients benefit from expert neurologists, today will provide a brighter future for all HD increase in funding from experienced and family-focused care,” said Louise psychiatrists, social workers, therapists, counselors families.” said Louise Vetter, HDSA’s President last year. Vetter, HDSA’s President & Chief Executive Officer. and other professionals who have extensive expe- and Chief Executive Officer. “The recognition of new Centers of Excellence means rience working with families affected by HD and that more families will benefit from comprehensive who work collaboratively to help families plan the care and the life-changing treatment and research best HD care program throughout the course of the opportunities that drive us forward towards finding disease. Applications to become an HDSA Center of a cure for this disease.” Excellence are open to all clinics in the United States who share HDSA’s commitment to high-quality, The HDSA Centers of Excellence provide an expert comprehensive care and access to clinical research.

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THE STARS SHINE AT FREEZE HD GALA MORE THAN On Saturday, September 28th, four hundred guests $10,000 to support this wonderful night! gaming equipment and so much more! Freeze HD Special Thanks To Our th attended the 5 Annual Freeze HD at Avalon “The 5th Annual Freeze HD was the largest was supported by a wonderful event committee $420,000 Freeze HD Gala Sponsors Hollywood where more than $420,000 was raised that included Rachel McAdams, Jason Ritter, fundraising event ever hosted to support our 9 10 to support HDSA’s fight against Huntington’s Jaime King, Marianna Palka, Simon Helberg, Bryce WAS RAISED organization and we couldn’t have done it without GOLD SPONSORS disease. This star-studded event was hosted by Dallas Howard, Kenneth and Diane Mayfield, TH our incredible honorees, Scott and Kelsey Porter, AT THE 5 • Genentech actor Lamorne Morris and honored Kelsey and Blake and Jenne Coler-Dark, Brian Logan Dales, sponsors, event committee and everyone who • The Ferrell-Paulin Scott Porter. Some notable guests included Seth Matt and Courtney Hamilton, Jamie Linden, Justin attended as well as bid on the amazing auction ANNUAL Family Foundation Green, Topher Grace, Jason Ritter, Katherine items,” said Louise Vetter, HDSA’s President and and Kate Miner, Elliott and Tracy Owen, Carly McNamara, Marianna Palka, Kate Miner, Rachel Ritter, Nancy Morgan Ritter, Tyler and Lelia Ritter, FREEZE HD 14 Chief Executive Officer. “Thank you everyone — SILVER SPONSORS Bilson, Wilson Bethel, Jaime King and Jason Ziven, Angelique Cabral, Jaylen Moore and this was truly a showcase of the extraordinary GALA 15 • Bel Air Internet Allie LaForce. The event featured incredible live Britt Logan, Gentille Koosed, Kyle Newman, Adam 13 strength of the HD community.” • Cambium Networks performances from Brian Logan Dales and The Seid, Jessica Cain, Emily and Jamie Rodgers and • Keyes Real Estate Dales Hotel, Tyson Ritter from The All-American The incredible auction included exciting trips, Alana Yankowitz. 11 • Marron Foods Rejects, DJ Skee, DJ Prophet, Emma Hutton, Leslie signed TV/movie & sports memorabilia, 12 • Wave Life Sciences Powell and Scott Porter. Actor Will Ferrell donated once-in-a-lifetime experiences, exclusive tickets, • Winncom Technologies

BRONZE SPONSORS • After Hours Theatre Company • Amberlee Colson • California Horizon Pool & Spa 16 • Educated Nannies • Fake Empire • Ferrandino & Son, Inc. • Freemark Financial LLP • Robert Kirkman • Rumble Riot Pictures 18 • Temple Hill Entertainment 17 • The MBS Group 1 2 3 4 5 • The O.C.D. Experience • Wilson Bethel 1. Will Ferrell 2. Ashley Hinshaw and Topher Grace 3. Freeze HD Introduction 4. Jason Ritter 5. Scott Porter, Jaime King, Rachel Bilson and Wilson Bethel share a playful moment 6. Kate Miner and • William Morris Endeavor Briana Lane 7. Todd Grinnell 8. Jaylen Moore strikes a pose 9. Marianna Palka and Kate Miner share a laugh 10. Tyson Ritter 11. HDSA President & C.E.O., Louise Vetter with Freeze HD Gala honorees, Scott and Kelsey Porter 12. Freeze HD Gala host, Lamorne Morris 13. Jaime King 14. Rachel Bilson 15. Katherine McNamara 16. Seth Green and Clare Grant 17. Brian Logan Dales performs 18. Rachel McAdams 19. Freeze HD Gala honorees, Scott and Kelsey Porter address the guests 19 4 5 HDSA FILMS DEBUT ‘ALL IN TO END HD’ STARTING A FAMILY AMID HD CONCERNS TM In 2019, HDSA produced three powerful short films CASINO NIGHT WITH GUILLERMO GILLETTE TREO RECEIVES POSITIVE DISCUSSED ON NBC’S THE TODAY SHOW — Carol & Richard: A Love Story, The Hodgsons, The ‘All In To End HD’ Casino Night took place REVIEWS FROM HD FAMILIES HD facts and statistics provided by HDSA were and The Vaughns. All were directed by Rae Maxwell. on Saturday, June 1 at the Continental Club in featured on The Today Show in February when our The films are featured on HDSA’s YouTube Channel downtown Los Angeles. The evening featured This year, HDSA partnered with Gillette dear friends, TNT basketball reporter Allie LaForce and and each has received thousands of views. a speakeasy venue setting, live music from jazz to test a new product with HD caregivers. husband Joe Smith of the Houston Astros appeared for singer Andrea Miller, various gaming tables and The Gillette TREO™ razor features a built-in an exclusive interview. The couple shared their concerns At right, a scene from The Hodgsons, produced by an appearance from emcee Guillermo from Jimmy shave gel and is the first razor specifically about starting a family since Joe’s mother suffers from HDSA Films. designed to shave someone else. Designed HDSA Films Kimmel Live! The event successfully brought in 125 HD. Dr. Erin E. Furr-Stimming, a neurologist from the guests and raised $35,000 for our HD community with caregivers, for caregivers, TREO has a University of Texas HDSA Center of Excellence, whose safety comb that NBC Universal — all while engaging new committee members, team was recently awarded a Clinical Research TNT’s Allie LaForce and the Houston Astros’ Joe Smith helps protect the ‘HERE’S THE DEAL’ VIDEO SERIES SEPTEMBER 18: potential sponsors and local influencers for next year. Pilot Grant by HDSA, also appeared. appeared on NBC’s The Today Show. HDSA’S FOUNDER’S DAY OF GIVING skin and prevent HDSA’s Jennifer Simpson, LCSW continues clogging, an to host Here’s The Deal, an online video On September 18th, HDSA ergonomic handle series that features insightful launched The Founder’s Day for great comfort COMEDIAN ROD MAN HOSTS HD conversations with members of the of Giving to pay tribute to and control, and a FUNDRAISER — ‘MAIN STREET LIVE’ HD community. This past year’s our founder, Marjorie special non-foaming Comedian Rod Man who comes from an HD family, topics included Isolation, Family Guthrie. It was on on gel that hydrates hosted his annual talent show — Main Street Live — Planning, Caregiving While Positive September 18, 1967 our the hair with no in Georgia to raise funds and awareness for HD. and Parenting a Child With JHD. organization was founded. water required. Rod has appeared on NBC’s Visit HDSA’s YouTube Channel to watch In just its first year, HDSA looks Last Comic Standing. the entire Here’s The Deal series. Founder’s Day of Giving to continue this This year his charitable raised more than great partnership Charlotte Reicks (left) and Marie Nemec event raised more $10,000 to support to bring useful than $3,000 to support the first-time attendee HDSA Jazz singer Andrea Miller products to ‘BIKE FOR THE CURE’ FEATURED HDSA’s mission. Convention Scholarship Program. Thank you! and Guillermo Rodriguez IN BICYCLING MAGAZINE from ABC’s Jimmy Kimmel HD families. Live! You can learn After 20 years and 20,000 miles, Bike for the Cure was Comedian, Rod Man more about the featured in Bicycling magazine. Over the past 20 years, Gillette TREO Charlotte Reicks age 83, and Marie Nemec, 74, rode and Rachel Nichols are among many more CELEBRITIES RAISE HD AWARENESS razor by visiting more than 20,000 miles for Huntington’s Disease celebrities who continue to generate much-needed Gillette.com/ Society of America. They crossed 48 of the 50 con- Seth Green, Jason Ritter, Marianna Palka, Kate HD awareness through #LetsTalkAboutHD TREO. tiguous states, connecting people and communities Miner, Jaime King & Rachel Bilson, Nyambi public service announcements. while growing their mailing list — their main source Nyambi, Wilson Bethel, Scott Michael Campbell of donations. As of December, 2019 they have raised over $743,000 to fight HD! 6 7 HDSA’S 34TH ANNUAL CONVENTION IN BOSTON SETS ATTENDANCE RECORD The 34th Annual Huntington’s Disease Society of affected by Huntington’s disease,” said Louise Boston, which led to the carnival-themed Welcome The education tracks were jam-packed with As is tradition, the Convention closed with the The 34th Annual HDSA Convention America (HDSA) Convention was held in Boston, Vetter, HDSA’s President and CEO. “With the Reception. During the Opening Ceremony on Friday workshops from world-class speakers, including emotional candle lighting ceremony paying tribute to was made possible by the generous Massachusetts on June 27th-29th at the Boston incredible advancements in HD research and care, morning, Dr. Jang-Ho Cha, Dr. Marcy McDonald Nobel Laureate Dr. Craig Mello who delivered the the entire HD community. support of all its sponsors: Marriott Copley Place. With 1,225 guests, the HD families are engaged and invigorated to go out and Cheryl Sullivan-Stavely led a moving panel Keynote Address during Saturday’s Research Convention set a new attendance record breaking and do something. We could not put an event discussing the dynamic relationship between Forum. Also during the Research Forum, Dr. Ed PRESENTING SPONSOR the previous record (1,046) in Los Angeles, of this magnitude together without the help of research and HD families. Wild and Dr. Jeff Carroll from HDBuzz gave an • Genentech th • Teva Pharmaceuticals California held last year. The 35 Annual HDSA our amazing sponsors who we are grateful to Friday afternoon featured the Cheers-themed in-depth and entertaining update on HD research Convention will be held in New Orleans, Louisiana call our friends in the fight against HD.” VolunCheer Luncheon where the Society cheered and promising clinical trials entitled “HD Avengers PLATINUM SPONSOR th th on June 4 -6 , 2020. The festivities kicked-off on Thursday with the the extraordinary efforts of the HDSA volunteers. — Endgame?”. • Lundbeck “The 34th Annual HDSA Convention not only set a Team Hope Walk through the historic streets of Friday night’s HDSA National Youth Alliance The Convention culminated with Saturday new attendance record, but it also confirmed the Talent Show raised more than $35,000 for the night’s elegant Gala Reception where HDSA GOLD NONPROFIT SPONSOR momentum of the NYA Scholarship Fund which sends children honored individuals and groups for outstanding HDSA 2019 NATIONAL AWARDS • CHDI Foundation / Enroll-HD HD community and young adults impacted by HD to the achievements with the HDSA 2019 SILVER SPONSORS that is actively HDSA Convention each year. National Awards (see list at right). Woody Guthrie HDSA Excellence • Ionis working to Advocacy Award in Care Award • uniQure improve the Seth Rotberg and Dr. Samuel Frank Therese Crutcher-Marin lives of Marjorie Guthrie Award BRONZE SPONSORS everyone HDSA Youth Award Patricia McLane and • MassMutual SpecialCare Hallie Kile Morgan McLane • Voyager Researchers of the Year HDSA Person of the Year • Wave Life Sciences Dr. Darren Monckton and Alexandra Boothby FRIEND SPONSORS Dr. Vanessa Wheeler E.J. Garner, HDSA Board of • Archcare HDSA Chapter of the Year Directors Chair (l.) and Louise • BioTek reMEDys HDSA New Jersey Chapter Vetter, HDSA President & • Broda CEO (r.) present Alexandra HDSA Affiliate of the Year Boothby HDSA’s 2019 • Next Step Healthcare HDSA Connecticut Affiliate Person of the Year Award. • PTC Therapeutics • Sanofi Inaugural • Spark Therapeutics Long-Term • UMass Medical School Care Award • Vaccinex Sitrin Health • WilmerHale Care Center

8 9 HDSA ADDS THREE TRUSTEES TO NATIONAL BOARD HDSA’S NYC MARATHON TEAM RAISES MORE THAN $133,000 The Huntington’s Disease Society of America is Jay Hughes is MGIC’s Executive Vice President of Jonathan Klein is Chair of the Mergers and HDSA’s 2019 New York City Marathon team excited to welcome three new members to the Sales and Business Development. He began his Acquisitions Group at DLA Piper in New York City. featured twenty-five runners from twenty different HDSA’s 2019 New York City Society’s national Board of Trustees. career with MGIC in 1987 as an Account Executive He concentrates his practice in the areas of mergers States across the US all willing to take on 26.2 miles Marathon Team: in the northern Virginia market. After moving to and acquisitions, private equity, corporate finance, to support the fight against HD.To date, the team Wendy Erler is the Vice President of STAR and Patient Mark Adler, Kari Baker, Jennifer Bandley, California in 1998 to assume the role of Sales securities matters and restructuring for both raised more than $133,000 finishing in the top 18 Insights and Advocacy at Alexion Pharmaceuticals Madeline Bolton, Michael Boyle, Aly Boyle, Manager, Jay was promoted to Vice President- domestic and international clients. Jonathan has among all NYC Marathon Charity Partners! where she is responsible for building an enterprise Molly Corse, Samantha Cox, Carlos Fonts, Managing Director in 2001 covering MGIC’s represented US and foreign clients in connection holistic solution model to gather and incorporate Jonathan Harrison, Emily Lefler, Pacific Region. In 2014, Jay accepted the position with cross-border acquisitions, divestitures, joint patient insights throughout the drug development Janine Lind Trembicki, Paula Lintz, of Senior Vice President, Sales and Business ventures, private financings, loan restructurings and NEW LOOK, NEW RESOURCES and commercialization processes. She leads the Jessica Marsolek, Erin Milek, Jessica Parker, Development and relocated to Milwaukee, workouts and general corporate matters. Jonathan AT HDSA.ORG Global Patient Advocacy function and has responsibility Alycia Patty, Carolyn Plaza, Ricardo Puente, Wisconsin. In his current role as Executive Vice received his J.D. from Georgetown University Law the advocacy team. Previously, Ms. Erler was on the Craig Robinson, Amber Rutledge, Michael Shea, In January HDSA released a new website President of Sales and Business Development, Jay Center where he graduated cum laude and his B.S. executive leadership team at Wave Life Sciences Rod Thompson, Kristen Westdorp and Sara Witzl. design with improved navigation, Google where she led Patient Advocacy and Commercial. is responsible for MGIC’s five regions, our National from Cornell University. Translate and other exciting new features Wendy graduated from Miami University and Account relationships and Marketing. He holds “We are thrilled to welcome Wendy, Jay and found at HDSA.org! earned her MBA from St. Joseph’s University. a Bachelor of Science degree in Finance from the Jonathan to HDSA’s Board of Trustees,” said Villanova School of Business. Louise Vetter, HDSA’s President & Chief Executive COUNTRY STAR CHRIS STAPLETON Officer. “Their diverse skills and experiences will SUPPORTS HDSA help contribute to the Society’s ability to achieve After being introduced to HDSA’s rich ‘THIS WEEK IN HD RESEARCH’ BLOG its mission and support families affected by HD.” musical history and incredible mission, singer- HDSA’s Leora Fox, PhD provides In other Board-related news, Kamran Alam was songwriter Chris Stapleton donated $5,000 weekly updates in the This appointed Treasurer and Jenne Coler-Dark will to improve the lives of people with HD and Week In HD Research blog. assume the role of Secretary. Bill Klein and Jennifer their families. Dr. Fox keeps you up-to-date Leyton have successfully fulfilled their six-year term on HDSA research activities, as integral members of HDSA’s Board of Trustees. recently published work about We are grateful for the exceptional volunteer Huntington’s disease, historical leadership that guides our mission work! Clockwise from left: Craig Robinson, moments in HD research and more. Follow Samantha Cox, Kari Baker, and Janine Lind Trembicki the blog at HDSA.org/blog

Special thanks to the accounting firmEisnerAmper Wendy Erier Jay Hughes Jonathan Klein for being the team’s Presenting Sponsor. The team’s fastest runner was Craig Robinson who finished FOLLOW US! HDSA.ORG with an impressive time of 2:34:55 — good enough facebook.org/HDSofA @HDSA for 118th overall! The team’s top fundraiser was Kari Baker from Florida who raised more than $11,000! HDSANational HDSA.org/Youtube

10 117 HD LEGACY — INSPIRATIONAL RUNNER SUPPORTING HDSA CHARITY NAVIGATOR AWARDS HDSA 4 STARS — THE GIFT TO FUTURE GENERATIONS HIGHLIGHTED IN RUNNER’S WORLD MAGAZINE ITS HIGHEST RATING — FOR THE FOURTH CONSECUTIVE YEAR

In response to a growing need to study exactly what Gabby McNary who has tested positive for the HD gene, ran the Chicago “We are proud to announce Huntington’s Disease Society Huntington’s disease (HD) does to the human brain, Marathon (her first) to honor her mother who she lost to the disease five years of America has earned our fourth consecutive 4-star rating. HDSA and CHDI Foundation announced a new collabo- ago. She also raised over $4,000 for HDSA. Her inspiring story was featured This is our highest possible rating and indicates that your ration in 2019 called HD LEGACY to support brain and in the October 14 edition of Runner’s World. organization adheres to sector best practices and executes other organ donations from families affected by HD who its mission in a financially efficient way.Attaining a 4-star would like to make a vital contribution to research. rating verifies that Huntington’s Disease Society of America This new program encourages the donation of brain and exceeds industry standards and outperforms most charities in other tissues from HD gene-expansion carriers, at-risk your area of work. individuals, and healthy family controls. The Harvard Only 20% of the charities we evaluate have received at least 4 consecutive 4-star Brain Tissue Resource Center (HBTRC) will assist evaluations, indicating that Huntington’s Disease Society of America outperforms interested most other charities in America. This exceptional designation from Charity Navigator families sets Huntington’s Disease Society of America apart from its peers and demonstrates throughout the to the public its trustworthiness.” process, and families will Michael Thatcher, President & CEO, Charity Navigator bear no costs associated with the donation. “The decision to donate one’s body to science is an amazingly selfless act,” saidLouise Vetter, President and CEO at HDSA. “Participating in HD LEGACY is truly a HDSA LISTED IN TOP 10 VOLUNTEER OPPORTUNITIES gift for future generations. The brains and other organs IN NEW YORK CITY BY GREAT NONPROFITS donated through this program will leave a ‘legacy’ of hope, inspiring new knowledge that will someday stop A woman whose mother was diagnosed with HD in its tracks.” Huntington’s disease forwarded the following comment to greatnonprofits.org about HDSA: “Science still has a quite rudimentary understanding of how the human brain works,” said Robi Blumenstein, “I am a daughter whose mother was diagnosed with HD, President of CHDI Management, Inc. “Researchers need which I knew nothing about until I found this organization. It more brain donations from HD-affected individuals to not only answered the billions of questions I had, but also provided me with emo- better understand how HD affects the human brain, what tional support from now friends. It gives me a sense of security knowing I am not the happens to the different cell types and circuits that make only person in the world going through this. This organization in a way saved my life. up the brain over time. HD LEGACY will give us new I was overwhelmed and ready to give up and they would NOT let that happen. For insight into how to intervene therapeutically.” Gabby McNary comforts her Mom. that I will always be truly thankful. This is an organization that helps people in every way possible. Thank you for all you have done and continue to do every day. I will always be a proud volunteer HDSA.” 12 131 2019 REGIONAL UPPER MIDWEST REGION GREAT LAKES REGION NEW YORK / NEW JERSEY REGION NEW ENGLAND REGION Twin Cities Team Hope Walk New York City Hudson Valley Minnesota Chapter Team Hope Walk Team Hope Walk $42,400 Greater New York Chapter New York/New Jersey EVENT HIGHLIGHTS $116,300 Region South Dakota $83,800 Team Hope Walk Celebration of Every year, HDSA’s PACIFIC REGION South Dakota Chapter Hope Dinner 50+ Chapters and $37,100 Albany Chapter $90,835 Affiliates host more Iowa Hoop-A-Thon Tour Sparkle Celebration Chicago Iowa Chapter of Hope Celebration of Hope than 300 events $27,000 Northeast Ohio Chapter Great Lakes Region $185,000 $129,900 across the country Illinois Team Hope Walk New England Region Boston Team Hope Walk thanks to the amazing Illinois Chapter Celebration Massachusetts Chapter $103,800 of Hope Gala $52,650 leadership of HD New England Region $62,500 Tewksbury Team Hope Walk families & volunteers. Massachusetts Chapter $48,000 Here are just a few! MID-ATLANTIC REGION San Diego San Francisco Los Angeles Team Hope Walk Team Hope Walk Team Hope Walk Team Hope Walk Philadelphia Special thanks to our San Diego Chapter Pacific Region Los Angeles Chapter Chesapeake Affiliate Team Hope Walk $65,000 National Team Hope $115,000 $65,000 $32,300 Eastern Pennsylvania Chapter Sponsors Manassas $30,000 SOUTH CENTRAL REGION Team Hope Walk Mid-Atlantic Region $30,797 Fairways for Hope Rocky Mountain Chapter $38,320 SOUTHEAST REGION Denver Team Hope Walk Rocky Mountain Chapter $34,700 21st Annual Cure HD 28th Annual Cure HD Golf Tournament Triathlon/Duathlon/ Anita Sabedra Tennessee Chapter Aquabike 2019 Team Hope Walk $124,129 South Florida Chapter South Central $64,275 Region $28,100 Atlanta Team Hope Walk Georgia Chapter $38,203 14 15 RESEARCH FUNDING PROGRAMS

2019 HUMAN BIOLOGY PROJECT FELLOWSHIPS Highlights from Previous HD Human Biology Project Fellowships

Madeleine Sharp, MD Marina Papoutsi, PhD Richard Hickman, MD 2016 Human Biology Fellow, 2017 Human Biology Fellow, 2018 Human Biology Fellow, Postdoctoral Research Fellow, McGill University Postdoctoral Researcher, University College London Neuropathology Fellow, Columbia University The overall goal of Madeleine’s project was to Marina is a skilled statistician whose project Richard’s HDSA funding is allowing him to work uncover the mechanisms underlying common focused on the concept of “cognitive reserve,” the closely with Dr. Jean-Paul Vonsattel to learn the art cognitive and behavioral symptoms of HD. idea that intelligence, education, or an intellectually of HD brain banking, an invaluable skill that enables Danielle Larson, MD Osama Al Dalahmah, MD She designed tasks to detect subtle changes in stimulating lifestyle can be protective or delay researchers to use donated human brain tissue. His learning and memory, even in pre-symptomatic cognitive problems for people with the HD gene. In Northwestern University Columbia University HDSA fellowship also helped him to transition to an carriers of the HD gene. She exceeded her enroll- 2019 she completed an analysis showing that HD assistant faculty position at Columbia University. He Feinberg School of Medicine Dr. Al Dalahmah will examine ment goals with a total of 79 HD patients, some patients with higher levels of education or a higher is working with autophagy expert Dr. Ai Yamamoto Dr. Larson will study whether how astrocytes, the brain’s of whom were recruited at HDSA’s National IQ had a slower rate of decline in mental abilities. to explore how the brain’s waste disposal systems remote “telemedicine” visits support cells, change over the Convention. This work is impactful from the This knowledge could be applied to design stimulat- handle toxic huntingtin protein. Beyond potential with a neurologist could be just course of HD to become harmful perspective of early diagnostics as well as potential ing interventions, encourage young people in HD discoveries that could help harness the power of the as helpful as in-person visits for versus helpful. interventions. Certain cognitive tasks shown to families to follow intellectual pursuits, or to stratify cell’s clean-up crew to combat HD, the skills required an HD patient or family. create long-lasting improvements in memory and patients in clinical trials. to make use of precious brain donations from HD reward processing could potentially be powerful and families are highly sought after. In October of 2019, HDSA awarded five safe approaches to treating early changes in thinking. grants under the Society’s largest research initiative, the HDSA Huntington’s Disease Human Biology Project. These grants, totaling $575,000, represent HDSA’s patient-centric research focus which brings basic and clinical researchers together to facilitate Huntington’s disease science in Vilija Lomeikaite, PhD candidate Ricardo Mouro-Pinto, PhD Saul Martinez-Horta, MsC, the human condition. Applicants worldwide University of Glasgow proposed projects addressing HD through Massachusetts General Hospital Sant Pau Hospital, Barcelona and Harvard Medical School small clinical studies or donated human Vilija will work on improving Saul will examine what kinds the existing methods to detect Dr. Mouro-Pinto will focus on the of changes are happening in samples. This year’s winners hail from an increase in CAG repeats in biology of CAG repeat expansion the brain and blood when HD Scotland, Spain, and the United States. certain cells of the body and in hopes of developing drugs to patients begin to have difficulty 2016 2017 2018 brain during the course of HD. stop this process. with planning and memory.

16 17 2019 DONALD A. KING SUMMER RESEARCH FELLOWSHIPS 2019 BERMAN-TOPPER FAMILY HD CAREER DEVELOPMENT FELLOWSHIPS In April of 2019, four exceptional undergraduate students were awarded HDSA’s undergraduates who have committed their summers to training in an HD As part of HDSA’s commitment to developing the next generation of passion- Dr. Byrne is working under the guidance of Dr. Ed Wild at University College Donald A. King Summer Research Fellowships. These awards are named in research lab. Applications are accepted between December and March each year ate and innovative Huntington’s disease scientists, the Berman-Topper Family London. Her project will better prepare the field for future disease honor of Donald King, who served as HDSA Board Chairman from 1999 to 2003 and are reviewed by HDSA’s Scientific Advisory Board. The 2019 Donald A. King HD Career Development Fellowships provide up to $80,000 of funding per prevention studies by researching how early in life we can detect changes in and worked tirelessly to advocate for HD families until his death in 2004. In order Summer Research Fellows completed focused HD research projects under the year for three years to young scientists and clinicians who desire to make HD the neurofilament light (NfL) protein in persons with the HD mutation. to further meaningful discoveries about Huntington’s disease and ensure that supervision of senior scientists. part of their long-term career plan. These prestigious awards are made possible bright young scientists are steered towards the field, this program supports due to the generosity of the Berman and Topper families and CHDI Foundation. Dr. Caron is guided by Dr. Michael Hayden at the University of British Columbia. HDSA’s Scientific Advisory Board reviewed applications from researchers from He will investigate novel ways to enhance the delivery of huntingtin lowering all around the world, and HDSA was able to award two scientists the 2019 antisense oligonucleotides across the blood-brain barrier. Berman-Topper Fellowship — Dr. Lauren Byrne and Dr. Nicholas Caron.

Dr. Lauren Byrne and mentor Dr. Ed Wild at HDSA’s Convention in Boston.

Zach Cook Chloe LaRochelle Alexandra Putka Colleen Strohlein Brown University University of Central Florida Duke University University of Pittsburgh Zach spent the summer working Chloe worked under the guidance Alexandra worked in the laboratory Colleen spent the summer working with Dr. Marc Tatar, testing of Dr. Amber Southwell on a project of Dr. Audrey Dickey to elucidate in the laboratory of Dr. Robert effectiveness of drugs in a fruit fly investigating aggression in HD mice. how altered function of a protein Friedlander, to investigate whether (Drosophila) model of Huntington’s called PPAR contributes to HD phosphorylation of huntingtin disease. pathology. protein affects its localization to mitochondria. Dr. Nicholas Caron

18 19 RESEARCH ADVOCACY EDUCATION

YOUTH PROGRAMS SUPPORT & SERVICES DISABILITY PROGRAM 20 21 RESEARCH ADVOCACY BY GEORGE YOHRLING, Ph.D. BY JENNIFER SIMPSON, LCSW

goal. Recent success in gene therapy 2019 saw positive growth and chance in our and Cassidy (R-LA) in the Senate. The HD Parity for spinal muscular atrophy provides advocacy agenda! We were lucky to have Catherine Act (HR 2770/S 1476), is a bill that HDSA and a strong sense of optimism that this Finley of Thorn Run Partners join us in the fight advocates have been fighting to pass for a decade, approach could alter the course of to pass the HD Parity Act as the leader of our and we are working hard as we can to see this bill HD, too. Novartis also announced Washington D.C.-based advocacy efforts. In May, cross the finish line this Congress. In July, more than plans to launch a Phase 2 trial Representatives Kinzinger (R-IL) and Pascrell, Jr. 20 advocates from across the country joined investigating an oral huntingtin- (D-NJ) introduced the HD Parity Act in the House HDSA’s President and CEO Louise Vetter to lowering drug. alongside their partners Senators Gillibrand (D-NY) advocate with members of the Senate Finance A record number of HDSA Committee, asking them to advance the HD Parity Convention attendees in Boston, Act through committee. But we didn’t stop there. MA were treated to a presentation Chapter and Affiliate members, Center of Excellence by Dr. Craig Mello, directors, Social Workers and allies continued to Nobel Laureate and work throughout 2019, sending messages to discoverer of the Members of Congress, inviting other community To Keep our HD Momentum Going science that has members to join them in advocacy, as well as Cures Act of 2016 in an effort to better capture We Must Have Even Greater Goals enabled huntingtin coordinating in-district meetings. Advocates in prevalence and incidence data on key neurological Ohio, Indiana and New Jersey met with Senators In last year’s HDSA Research Report, I stated, “I know lowering trials to conditions on a national scale. Having accurate become a reality. Rep. Adam Kinzinger (R-IL) Rep. Bill Pascrell, Jr. (D-NJ) and Representatives right at home, engaging their information on the incidence and prevalence of that the HD families around the world will be ready Members of Congress, building relationships, and to answer the call for brave research volunteers to get Dr. Mello and Huntington’s disease would be invaluable in order to colleagues announced the putting human faces to the advocacy being done better direct clinical and support resources around the disease-modifying trials completed... quickly.” behind the scenes in D.C. The impact of these However, I could not have predicted just how quickly! formation of a new company country. HDSA continues to advocate with the CDC to that will try to treat HD. This is one meetings, contacts and relationships is incalculable, grow the NNCSS to include Huntington’s disease, and After years of waiting, in 2019 the HD community of dozens now currently pursuing HD and invaluable. better assess the impact of HD on U.S. families. was rewarded with a spree of potential disease- as a primary indication in their business plan. The inspiring scientific progress within the HD HDSA was also asked to take part in a neurological modifying clinical trials. With such anticipation and research community this year gives me confidence conditions summit held at the Centers for Disease demand, the Natural History and GENERATION- While the light of hope now shines brighter for all that 2020 will bring generations of HD families one Control on the growing National Neurological JENNIFER SIMPSON, LCSW is HDSA’s HD1 studies from Roche-Genentech, the Precision of us, much work remains before HD becomes a step closer to relief. Conditions Surveillance System (NNCSS). The Senior Manager of Advocacy and Youth Programs. HD2 trial from Wave Life Sciences, and the Signal thing of the past. That work requires the voices Sen. Kirsten Gillibrand (D-NY) Sen. Bill Cassidy (R-LA) NNCSS was developed as part of the 21st Century study from Vaccinex all completed US recruitment and the continuing participation of HD families. In Thank you for all you do! this year, at record speed. 2019, HDSA continued to enable families to learn about and participate in HD research, through HD While we await results from these studies, the HD Trialfinder, our research blog and webinars, support GEORGE YOHRLING, Ph.D. is HDSA’s research momentum continues. This year, uniQure of HD Buzz, the international Coalition on Patient Chief Scientific Officer and Chief Mission Officer began the first ever gene therapy trial for HD, and Engagement (HD-COPE), and more. several other companies are moving towards this

22 23 EDUCATION YOUTH PROGRAMS BY DEBRA LOVECKY, MS BY JENNIFER SIMPSON, LCSW

2019 was an exceptional year for HDSA In addition to outstanding programming in the 100 family members to each. HDSA thanks 2019 saw the expansion of HDSA’s stable of youth youth social workers. HDSA and the NYA brought workers, to utilize them for peer outreach and educational efforts. Thanks to a generous areas of care, advocacy and youth, several larger Genentech for their generous unrestricted support programs, rolling out our Youth Social nearly 90 young people out to our youth retreats in support. Regional Leads also took center stage unrestricted educational grant from Genentech, conferences received a clinical trial update from educational grant which made these events Worker Pilot Program and the integration of these 2019, providing them with information from experts during a 2019 advocacy trip to Washington D.C., HDSA was able to support 59 educational events Genentech as well as information about advances possible. two social workers into the world of youth work. at HDSA Centers of Excellence, support from social using their stories to influence leaders to advance that ranged from full day in HD research. Every attendee In the area of professional education, HDSA In 2019, the HDSA National Youth Alliance (NYA) workers, and integrated improv and art therapy legislation important to the HD community. conferences to half-day received an HDSA information launched a new free three year continuing held their four youth retreats around the country, workshops. Here’s to more incredible experiences, growth and meetings to support packet that contained valuable education course in partnership with the American returning to Pittsburgh and New Orleans and The NYA also funded more than 60 young people to support for young people impacted by HD in 2020! group guest speakers. materials about support groups, Occupational Therapy Association (AOTA). We introducing Chicago and Sacramento as new sites. attend HDSA’s largest ever National Convention in This compares favorably HDSA Centers of Excellence and anticipate that it will be as well received as the prior As we look to 2020, expect more new cities to be Boston through full and partial scholarships. The with 2018 when HDSA chapters/affiliates in their region. three year course which awarded more than 2,200 added to our rotating sites for NYA Youth Retreats. NYA also supported their Regional Lead Program, supported 49 educational events. Our thanks to the Almost 2,700 HD family members and healthcare With our Youth Social Workers joining us in Chicago JENNIFER SIMPSON, LCSW is HDSA’s Continuing Education Units (CEUs) to community- encouraging chapters and affiliates, as well as local Senior Manager of Advocacy and Youth Programs. creativity and budget savvy of our event organizers professionals attended these events with six based occupational therapy specialists. and Pittsburgh, young people can anticipate even social workers, support groups and youth social who used every one of our grant dollars wisely. multi-track state conventions attracting more than more support in 2020 as we bring on two additional In early 2020, HDSA will launch a new three year continuing education course with the American Society of Genetic Counselors (NSGC) that will provide the information community based genetic counselors will need to counsel and assist at-risk individuals contemplating HD genetic testing. HDSA was invited to present two posters at the annual Huntington Study Group meeting in Sacramento CA. Each poster focused on HDSA’s professional education efforts and partnerships with AOTA and Medscape (which awarded more than 5,000 CEUs to healthcare professional in 2018). As always, HDSA is pleased to have featured so many of our HDSA Center of Excellence staff in these continuing education efforts.

DEBRA LOVECKY, MS is HDSA’s Director of Educational Programs.

24 25 SUPPORT & SERVICES DISABILITY PROGRAMS BY ANNE LESERMAN, LCSW BY ALLISON BARTLETT, ESQ.

Ask a social worker! Families and many profession- Boston. This in-person educational event helps HD Many families find support and a listening ear at In 2019, HDSA unveiled its new disability program Security disability process, including eligibility including a Disability Booklet, Wellness Journal, and als ask HDSA supported social workers questions social workers provide the best and up to date HDSA in person support groups that are available in thanks to the addition of disability attorney, Allison criteria, work credits, and the 5-step disability Social Security Disability Application Tips. about genetic testing, long-term care placement, information on assisting HD families with their many states. Our on-line groups are open to those Bartlett, Esq., who specialized in the representation evaluation process. To date, Disability Chat has had If you have questions about Social Security locating resources in their community, accessing concerns. The national office continues to person a around the country. There are groups for caregivers, of clients with rare, chronic conditions, like Huntington’s 119 live views and 441 recorded views. HDSA has disability or general disability topics, please contact benefits and getting information and support about helpline for families and professionals and refers to those at risk for HD, those early in the course of disease, to navigate them through the complex also expanded the ‘Disability Benefits and HD’ Allison Bartlett, Esq. at 212-242-1968, ext. 218 the disease. Families find it comforting to talk with the chapter and regional social workers who their illness, parents caring for children with HD disability system. She works directly with families section (under Find Help, Healthcare & Future someone who understands the complexities of HD. respond to thousands of requests. Social workers and those in HD families that will not get HD. and social workers to provide advice, assistance, Planning) on its website to include new disability also interact with professionals in their communities Groups help families understand and normalize the and resources for the Social Security disability resources. Several new resources were created for HDSA is proud of the services that we provide to ALLISON BARTLETT, ESQ is HDSA’s symptoms that can occur in HD persons. Caregivers process. We have assisted and provided educational families and social workers this year to help with the families throughout the country. Social workers in doing presentations to medical students, genetic Manager of Disability Programs. and HD persons can all benefit. Individual counseling resources to approximately 91 social worker Social Security disability application process, HDSA Centers of Excellence, chapters, regions and counseling students, behavioral health students, sessions are also requests and 222 other HD clinics assist families in their 47 states. law enforcement personnel, assist in educational offered through HD families via phone Our social work professional training brought 68 day events, provide in-services to long term care American Well® and email regarding social workers together prior to convention in facilities or other health agencies and participate in HDSA National Youth Alliance (NYA) retreats. (www. amwell.com), the Social Security a telehealth program. disability process and Many continue to take the appropriate next advantage of free steps for the disability counseling sessions application in the from mental health areas including: when professionals in their state that are knowledgeable to start the disability process, tips for submitting a about Huntington’s disease. disability application, health insurance, work credits Check out the HDSA website (www.HDSA.org) and and disability eligibility, and case specific disability use the Locate Resources button to find services in questions. your area. Ask a social worker! HDSA launched the Disability Chat Webinar Series in September 2019 to cover a wide range of disability topics for families and social workers. Disability chat webinars are hosted every other month and provide ANNE LESERMAN, LCSW is HDSA’s Assistant Director of Community Services. an in-depth look at different aspects of the Social

26 27 Please Join Us!

MISSION BOARD OF TRUSTEES STAFF LEADERSHIP To Improve the Lives E.J. Garner Louise Vetter of Everyone Affected by Chair President and Huntington’s Disease Chief Executive Officer and Their Families. Victor Sung, M.D. Chair-Elect Rosemary Coluccio VISION Arik Johnson, Psy.D. Chief Operating Officer A World Free of Past Chair Christopher Cosentino Huntington’s Disease. Kamran Alam Director of Marketing Treasurer and Communications Jenne Coler-Dark Nancy A. Rhodes Secretary Director of Field Development Dan Brennan Jamison Skala Director of Development Stacy Coen George Yohrling, Ph.D. Wendy Erler Chief Scientific Officer and Donald Higgins, M.D. Chief Mission Officer Save the Date! Jay Hughes Join us for the 35th Annual HDSA Convention Jonathan Klein, Esq. in the Big Easy at the Sheraton New Orleans Hotel. Teresa Srajer Leslie Thompson, Ph.D. JUNE 4-6, 2020, NEW ORLEANS, LA Daniel S. Vandivort Vicki Wheelock, M.D. Register today at HDSA.org/convention

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