2018 Annual Report Contents

Welcome Message 3

MDA is Changing with Changing Times 5

Innovations in Science 7

Innovations in Care 13

Meet our Partners 21

Partners in Progress 22

Strength in Numbers 23

MDA Board of Directors 27

Research Advisory Committees 29

Financial Report 31

Recognition and Accreditation 33

1 2018 ANNUAL REPORT 2 In 2018 many of our efforts re-dedicated our commitment medicine, clinical trials, A Message came to fruition with critical to the support of clinical regulatory science, emerging impact. Spinal muscular research initiatives. Since use of technology, and from Rodney atrophy (SMA) was added to its inception, MDA has newborn screening. We were the Recommended Uniform invested more than $1 billion honored to have Dr. Janet Howell, MD, Screening Panel (RUSP) for in neuromuscular disease Woodcock, director of the newborns. In 2018 we also research to uncover new Center for Drug Evaluation chairman, launched the neuromuscular treatments and cures. We and Research (CDER) at the Observational Research Data had 224 grants active during Food and Drug Administration MDA Board of Hub in 25 MDA Care Centers 2018 representing, a funding (FDA), provide the opening with plans to expand to 50 commitment of more than $58M. keynote address. Directors, and in 2019. And in December, We launched new educational the U.S. Food and Drug seminars and programming for In May, the American Society Lynn O’Connor Administration accepted an both healthcare professionals for Gene and Cell Therapy application for the first-ever and our community. (ASGCT) awarded MDA the Vos, president gene therapy treatment for a Sonia Skarlatos Public Service neuromuscular disease under We connected one-on-one with Award for 2019. The award and CEO, Priority Review status. MDA individuals and families recognizes a person or group while responding to more than that has consistently fostered RODNEY Muscular We began 2018 by dialoguing 20,000 inquiries received by our and enhanced the field of gene with key members of our National Resource Center. And and cell therapy through HOWELL, MD Dystrophy community — neurologists we made our annual Summer governmental agencies, public and families — to gain deeper Camp program, “the best week policy groups, public education, Association insight into their specific needs of the year” for many of our kids or non-governmental and respective experiences and families, even better by charitable organizations. We are living through in working on and living with offering families choices about unprecedented times in neuromuscular disease. where kids could attend. We are so grateful and inspired Learning from this important by our Care Center teams, neuromuscular disease. research led to two landmark In 2019, MDA has already seen sponsors, volunteers, and Our work, across all reports – “Understanding some big accomplishments. donors for their unwavering Neuromuscular Disease In April we held our first ever support of our important mission. of MDA, is paving a Care” and "ONEVoice." combined Clinical & Scientific new path forward in Conference, themed “Progress Thank you! terms of understanding The Healthcare Partnerships in Motion,” in Orlando Fla. Team was established in Clinicians, scientists, neuromuscular disease 2018 with the charter to build policymakers, nonprofit, and and enabling life-changing synergistic partnerships in the industry leaders convened innovations in research, healthcare industry, starting for a dynamic and informative with pharmaceutical and biotech five days. With more than Rodney Howell, MD treatment, and care. companies and then expanding 1,250 attendees, 23 sessions, Chairman, Board of Directors over time to other healthcare 136 presentations, and 300 Muscular Dystrophy Association verticals including durable posters, this was MDA’s largest LYNN O’CONNOR medical equipment (DME), conference ever. Also in retail and specialty pharmacy, attendance were 30 exhibitors, VOS diagnostics, and technology. five companies on Technology Lynn O’Connor Vos Row, and 13 nonprofit partners President and CEO MDA has deepened its roots in in our Patient Advocacy Muscular Dystrophy Association the science of neuromuscular Pavilion. The major themes of health and disease and the conference were genetic

3 2018 ANNUAL REPORT 4 Not long ago, supportive MDA-funded breakthroughs MDA is care and mobility aids were include drugs for Duchenne Our Mission: Changing all that could be offered to muscular dystrophy (DMD), Transformation patients with NMDs. But periodic paralysis, Pompe with the treatment and care disease, and SMA. Through landscapes have begun to Changing change dramatically for many And it’s not just about new Innovation people living with NMDs. therapies. Thanks to research Times Recent breakthroughs in and advocacy efforts of As the largest and longest- genetic medicine, along MDA and its partners, new established organization Fighting neuromuscular with other discoveries, are diagnostics and screening for people with NMDs, MDA disease (NMD) today is accelerating the development policies enable earlier works tirelessly on the NMD about bringing together of new therapies, including detection and treatment. community’s behalf. We’ve the best and brightest some that not just address The goal: prolonging muscle done so for nearly seven decades. Now, with growing medical minds in search symptoms but also — for the function and lives. first time — directly modify scientific knowledge and of a cure. the underlying disorders. At an explosive pace, advances the expansion of improved in care, communication, and treatment options come new Support enables MDA to collaboration are now upending ways for us to transform the More than fund leading research teams long-held assumptions about lives of people with muscular working toward breakthrough people with NMDs; never have dystrophy, ALS, and related therapies that can have so many possibilities existed neuromuscular disease a life-changing impact for a community that once through innovations $1 billion on patients. faced progressive disability and in science and innovations premature death. While in care. invested to date NMDs still remain extremely challenging to live with and treat, today more people than ever in accelerating MDA supported before can look forward to lives 224 research with potential unimaginable just the development projects in 2018. a few years ago. of therapies Yet a tremendous amount of work remains to be done. and cures.

5 2018 ANNUAL REPORT 6 Research We are therefore bringing Innovations new resources and skills in Science MDA is the largest source to rapidly transfer this of funding for neuromuscular exponentially growing Support for MDA's disease (NMD) research body of knowledge from research enables us outside the federal government. laboratory to clinic, where it can change lives to fun teams working Therapies as never before. RESEARCH toward breakthrough therapies, which may Research supported Parts of MDA’s new GRANTS IN 2018 have a life-changing by MDA is directly role include: impact on patients. linked to life-changing therapies across multiple • Working with investigators neuromuscular diseases. on new, improved clinical Importantly, MDA’s network trial designs and real- model works across the world evidence 224 64 42 full spectrum of NMDs to cross-pollinate ideas and • Helping scientific partners GENERAL ALS DMD/BMD cross-link disease-specific view, track, and interpret RESEARCH developments to potential unbiased and up-to-date applications that help the data to make fully entire NMD community. informed decisions about future innovations 10 14 2 SMA LGMD POMPE As knowledge about NMDs • Convening clinical has vastly expanded, so has stakeholders to change the complexity of diagnosis standards of care as and treatment. Science has 11 15 12 new approaches uncovered scores of specific become available FSHD DM CMD genetic dysfunctions underlying NMD subtypes. 13 CMT

ALS = amyotrophic lateral sclerosis; DMD/BMD = Duchenne muscular dystrophy/Becker muscular dystrophy; SMA = spinal muscular atrophy; LGMD = limb-girdle muscular dystrophies; FSHD = facioscapulohumeral muscular dystrophy; DM = myotonic dystrophy;CMD = congenital muscular dystrophy; CMT = Charcot- Marie-Tooth disease.

7 2018 ANNUAL REPORT 8

MDA launched the Innovations MOVR (neuroMuscular Healthcare in Science ObserVational Research) Partnerships Team Data Hub as a transformative Big data is a key part platform, combining MDA’s In 2018, the Healthcare Partnerships Team was of MDA’s evolution. Care Center Network with a As the largest centralized created to develop relationships with leading state-of-the-art information- data hub for multiple pharmaceutical and biotech companies in the management system. neuromuscular diseases, NMD space. In addition, the team established MOVR aggregates clinical, and now manages an agency-caliber healthcare genetic, and patient- communication and educational platform. This reported data across broad includes offering accredited continuing medical communities of healthcare education (CME) programs as well as community providers, researchers, and educational initiatives. industry partners that will lead to rapid developments The team also manages MDA’s annual conference, in patient care, treatments, which in 2018 brought together more than 500 and cures. medical and scientific neuromuscular experts. The conference focused on providing opportunities for MOVR will be an unparalleled physicians and allied health professionals to learn one-stop resource, an asset about new approaches and techniques for clinical to researchers probing for management, hear about the latest information answers that could unlock regarding clinical trial results, and engage in new NMD science, and a dialogue and networking among peers. powerful force for improved coordination of care The team is responsible for publishing Quest, for individuals. MDA’s quarterly publication, which has nearly 800,000 touch points annually.

The Healthcare Partnerships Team exists today to support the tremendous growth being seen in drug development and the critical educational needs of our community in response to new therapies and treatments being brought forward through R&D.

9 2018 ANNUAL REPORT 10 Genetic medicine Indeed, that several Innovations experimental gene therapies in Science MDA has contributed greatly are in advanced clinical trials to the field of muscle disease in our diseases is the result MDA has long and toward landmark of decades of effort from a appreciated that gene research advances, including large community of partners —including families, donors, therapy, by targeting the identification of the first human disease-causing researchers, clinicians, the very root cause of the gene, as well as the first and members of industry. disease, has tremendous human trial of gene transfer therapeutic promise for a muscle disease. Industry recognition in a wide array of neuromuscular diseases. For several decades, In 2019, MDA was granted MDA-supported researchers the Sonia Skarlatos Public have discovered the Service Award from the gene-causing mutations for American Society for Gene many other neuromuscular & Cell Therapy (ASGCT).* disorders, developed and refined gene delivery tools and methods, and established protocols for safe and effective gene therapy clinical trials. This was achieved by robust and rigorously-reviewed MDA grant programs aimed at driving the field of gene therapy forward, as well as MDA taking a leadership role in continually raising awareness and convening key experts to address challenges.

* This award recognizes a person or group that has consistently fostered and enhanced the field of gene and cell therapy through governmental agencies, public policy groups, public education, or non-governmental charitable organizations.

11 2018 ANNUAL REPORT 12 The cornerstone of MDA’s healthcare specialists NATIONAL CARE Innovations Innovations in Care conveniently sited at one in Care programming is MDA’s location. In a single day, CENTER NETWORK National Care Center patients can see multiple MDA programs and Network, a system of healthcare providers who services enrich the multidisciplinary medical work together to ensure clinics across the US at coordinated individual lives of people with more than 150 top medical care for every patient neuromuscular disease. institutions. These centers to best fit their specific 70,000+ 200+ CONVENIENT conduct 70K+ medical visits needs. Highly trained MDA ANNUAL annually for individuals Care Specialists serve PATIENT VISITS LOCATIONS living with muscular as an important part of dystrophy, ALS, and related the care team, helping neuromuscular diseases. families navigate the Each of our MDA Care health system, answering Centers offers individuals questions, distributing MDA and families best-in- educational materials, 50,000+ class, comprehensive coordinating MDA services, INDIVIDUALS 2,000 CLINICAL care from an integrated and assisting with community HELPED multidisciplinary team of resource referrals. PROVIDERS

12,000 PEOPLE ENGAGED IN NMD RESEARCH

MDA Care Centers also serve as regional and local hubs of NMD research activity for clinical trials and natural history studies. As major gateways to the MOVR Data Hub — both as expert accumulators of NMD knowledge and as sites where this knowledge can be tapped firsthand— the centers perform a vital function in advancing care.

13 2018 ANNUAL REPORT 14 Now that we understand newborn screening programs We are committed to Innovations better the crucial role by adding tests for specific understanding the natural in Care genetics plays in NMDs, neuromuscular disorders so history of neuromuscular access to early screening, that all newborns with these disease. When babies are MDA has taken diagnosis, and treatment is conditions can have the best diagnosed early in life, it a leadership vitally needed. For example, possible chance at receiving allows the opportunity to we know that SMA is the the care and support learn more about how the position in promoting leading genetic cause of services they need as early disorders manifest and to newborn screening. death in infants in the US, as possible. obtain insights into how and that early identification early intervention affects and intervention are key to disease course. treating the disorder. Once babies with

Tactics to support newborn these conditions are screening include peer- identified via state reviewed journal articles, newborn screening convening coalition partners to promote federal programs, MDA engagement around Care Centers can congressional and regulatory play a key role actions, engagement with state policymakers to in confirmatory encourage implementation, diagnosis, the establishment of an treatment, and advocacy grant program, and partnering with long-term follow researchers to help them up and care. complete studies of new testing technology. In many cases, the follow-up The International Association care may be lifelong and, in of Fire Fighters and MDA some situations, the clinical are also collaborating on symptoms may not manifest a campaign to encourage until later in life. states to expand their

15 2018 ANNUAL REPORT 16 Innovations Camps Collaboration Advocacy amplified US. The MDA Advocacy via collective impact Conference brings together in Care Throughout our evolution, Key events such as MDA individuals and families living many of MDA’s most admired Muscle Walk, MDA Team As the group whose with muscular dystrophy, ALS, MDA is changing with efforts and activities have Momentum, and the MDA constituency spans a broad and related neuromuscular stayed constant. Our MDA Shamrocks Program continue a clear mission, an range of NMDs, MDA helps diseases to carry a unified Summer Camps remain to unite our community and focus and unify efforts at the message to Congress, and expanded vision, and places where, for a week, bring awareness to our legislative and regulatory beyond. Additionally, MDA a renewed focus to kids and teenagers with mission. As we expand our levels, advocating for changes keeps advocates informed meet the needs of a NMD can gain independence collaborative efforts with that can address needs across about key initiatives and community undergoing and have fun as they learn new partners, we continue these diseases. provides channels for vital life skills, like building to cherish and nourish a remarkable transition. advocates to take action confidence and learning our long-standing links to MDA engages more than through a monthly self-advocacy. Each camp is groups and individuals who 9,000 advocates, reaching newsletter distributed to staffed with dedicated health have helped make us who all 50 states across the 40,000+ individuals. professionals and trained we are today, including: camp volunteers who meet the medical and physical needs of each camper — all at no cost to families. REACHING 9,000 ALL ADVOCATES 50 MORE THAN A MONTHLY STATES ACROSS NEWSLETTER THE U.S 100,000 DISTRIBUTED TO INDIVIDUALS AND FAMILIES LIVING WITH MUSCULAR 40,000+ DYSTROPHY INDIVIDUALS

MDA is a leading voice in the We advocate for policies patient advocacy community that maximize opportunities in our work with policymakers for independence. We also to ensure that all individuals maintain an Accessible Air living with neuromuscular Travel Resource Center disease have access to because the ability to travel the healthcare they need by air impacts many aspects through comprehensive of life, including options for health coverage. employment, education, and whether you can get back and forth from a clinical trial or a specialist’s office that is far from home.

17 2018 ANNUAL REPORT 18 MYOPATHIES (e.g., POMPE) Innovations Medical and opportunity to learn from, be Neuromuscular community education inspired by, and share ideas in Care with experts from academia, Diseases ION medicine, government, CHANNEL MITOCHONDRIAL MUSCLE DISEASES We offer a broad and and industry. Addressed MDA has taken on a vastly expanding array of resources DISEASE expanded role in meeting and events expertly At the community level, we by MDA the evolving educational developed to respond to established MDA Engage, needs of healthcare the rapidly changing a flagship educational event 43+ providers, patients, treatment landscape. series that brings local DISEASES and families. high-impact educational Our resources for providing programs to the NMD PERIPHERAL MUSCULAR relevant medical education NERVE DYSTROPHIES community. Each of DISEASE to professionals are (e.g., DMD, DM, the Engage programs (e.g., CMT) FSH, LGMD unparalleled, and our incorporates multiple services and initiatives modules of interest, from MOTOR NEURON reflect our leadership in therapy development this area. We provide both DISEASES roundtables to disease (e.g., ALS, SMA) accredited continuing management to genetic medical education (CME) testing, designed specifically and non-CME programs. for community audiences. Each event also includes a Since 1950, MDA has served as the only national patient As the most comprehensive social element for families advocacy group supporting more than 43 neuromuscular neuromuscular disease and participants with the diseases and the communities affected by these conditions. meeting, in the US, our aim of strengthening the annual MDA Clinical & community and helping Scientific Conference attendees make provides a unique personal connections. Moving forward a community undergoing a remarkable transition — Recent changes in care and whose prospects for paradigms for neuromuscular transformation have never diseases have greatly been better. Join us in our intensified the clinical, revitalized efforts to create a informational, and advocacy new, better world for people needs of the communities we with NMDs. serve. MDA is changing with a clear mission, an expanded For more information, vision, and a renewed visit www.mda.org focus to meet the needs of

19 2018 ANNUAL REPORT 20 Meet our Partners Partners in Progress

We believe great impact We are grateful to the Continuing a 65-year began in 1954 when the cannot be made alone. following partners tradition of giving strength to organization committed by Together, with our whose efforts and the MDA community, 100,000 proclamation to support MDA dedicated firefighters hit until a cure is found, and the generous partners, generosity each helped the streets or storefronts organization’s unwavering we’re raising life- generate more than in 2018 with boots in commitment to MDA has changing funds and $3 million in 2018. hand asking pedestrians, remained strong to this day. awareness for families motorists, customers, and As MDA’s largest national with muscular dystrophy other passersby to donate partner, they have raised to MDA raising more than more than $650 million and related diseases. $22 million. The partnership for MDA to date. Since 1950, MDA has between the IAFF and MDA been proud to team up with caring corporations, organizations, and brands that generate tens of millions of dollars each year through creative Mansfield campaigns, sales As MDA’s largest corporate Harley-Davidson Motor promotions, and year- partner, CITGO Petroleum Company has put its round special events. Corporation has raised commitment to freedom and more than $240 million for independence into action MDA through a wide variety by supporting MDA since of fundraising events, 1980. During its more than including golf tournaments, 38-year partnership with sporting events, in-store MDA, the Harley-Davidson retail programs, and family of dealers, customers, social events. CITGO also employees, suppliers, and organizes special events H.O.G. chapters raised more in ; Lemont, Ill.; than $103 million to help Corpus Christie, Texas; save and improve the lives and Lake Charles, La., of MDA families. Go to where the company has https://www.mda.org/get- major operations. CITGO involved/meet-our-partners marketers and retailers throughout the country to learn more participate annually in the MDA Shamrock Program and other events.

21 2018 ANNUAL REPORT 22 Strength in Numbers

It takes a village to achieve CONSTITUENT SNAPSHOT: a mission as important as ours. When lives are at stake, 31,000 retail locations every voice, every dollar, and 55% MALE participating in MDA events every individual matters. Nearly 9,500+ local fundraising events MDA is proud to be a grassroots 65 organization with a national field offices reach. Although we’ve been 45% FEMALE Nearly 500 golf events and galas leading the fight against muscular dystrophy and with 35,000 participants related diseases for almost MOST COMMON DISEASES 70 years, we haven’t done AFFECTING MDA FAMILIES: it alone. CMT, ALS, MG, DM, DMD, SMA Nearly 600,000 unique monthly visitors to In decades past, people held mda.org — that’s about 7.2 million people a year backyard carnivals, participated in bike-a-thons, and donated 15% MILLENNIALS to the telethon. Today, that 350,000 hometowns across America incredible spirit of creativity, generosity, and unparalleled 1.5 million+ POWERFUL SOCIAL passion is as vibrant as ever MEDIA COMMUNITY in a new era of giving people with a and innovation. connection to muscular dystrophy reached through email

Nearly 1million touchpoints for readers of

Magazine & MDA’s Strongly blog

23 2018 ANNUAL REPORT 24 MDA Fundraising Muscle Walk MDA Muscle Walk MDA Team Momentum MDA Shamrocks and

other pinups Events MDA Muscle Walk is more Team Momentum is MDA’s MDA special events are than a fundraising walk — it’s endurance training program Each year MDA’s iconic a fun way for generous a life-changing event that that empowers individuals of St. Patrick’s Day fundraiser all athletic abilities to train Muscle Walk donors, event participants, strengthens families and is supported by more than communities. In 2018, about for a half or full marathon 20,000 retail locations across and dedicated volunteers 29,000 people participated while supporting families the country — including to join their communities in 140 MDA Muscle Walks, with muscular dystrophy. In supermarkets, convenience in raising vital funds and raising $6.1 million. Since 2018, MDA Team Momentum stores, restaurants, and awareness to support 2011, MDA Muscle Walk has — and its more than 725 other community-minded participants — raised more individuals and families raised more than $43 million businesses. The public also to help bring strength to life than $1.2 million to help joined in our first Green living with muscle- for the families we serve. families. Since 2014, MDA Day for MDA, which was debilitating diseases. Team Momentum has had held in conjunction with the To learn more, and to sign 3,591 participants and raised Shamrocks campaign. In Here’s a look at the up and participate, go to a combined $5.29 million. 2018, MDA Shamrocks, contributions they helped mdamusclewalk.org. including all pinups, raised raise to support individuals To learn more, and join a race more than $14 million, and event, go to mdateam.org and families and programs for more than 35 years, in 2018. partners, employees, and donors have helped raise more than $310 million. Your Way for MDA To learn more, go to Your Way for MDA is an online MDA Distinguished Events mda.org/shamrocks. platform that gives MDA supporters the opportunity More than 36,000 people to decide how they’ll raise participated in 330 galas, money for kids and adults golf tournaments and Topgolf in their community. In 2018, tournaments across the thanks to the public’s creative country in 2018. More than ideas, Your Way for MDA $22 million was raised raised $110,919. Since the through these signature program began in 2015, MDA events to help advance supporters have raised more MDA’s mission. than $500,000. To learn more, go to To learn more, and contribute mda.org/get- involved/ to Your Way, go to paticipate-in-an-event mda.org/get-involved/ fundraise-your-way

25 2018 OFFICERS 2018-2019 DIRECTORS

Board of Chairman, Board of Directors Stanley Appel, MD Louis Kunkel, PhD Directors R. Rodney Howell, MD Houston, TX Boston, MA Miami, FL Anjan Aralihalli Patricia Nazemetz Vice Chair, Board of Directors New York, NY Sleepy Hollow, NY Christopher J. Rosa, PhD New York, NY John Constantino, Esq. Mike Rowlett New York, NY Farmers Branch, TX Secretary Charles D. Schoor, Esq. Benjamin Cumbo III Mark Smith Valley Village, CA Upper Marlboro, MD Houston, TX

Treasurer Steve Farella John Tognino Victor Wright New York, NY Ardsley, NY New York, NY Steve Felsher Kristine Welker New York, NY Hartsdale, NY

Daniel Fries Gene Williams New York, NY Cambridge, MA

Ankur Ghia Don Wood Washington DC Odessa, TX

Honorable Brad Henry Lilian Wu, PhD Norman, OK Armonk, NY

Dave Hutton Valencia, CA

27 2018 ANNUAL REPORT 28 Research 2018 RESEARCH ADVISORY MDA VENTURE PHILANTHROPY Advisory COMMITTEE MEMBERS ADVISORY COMMITTEE MEMBERS Committees Stanley Appel, MD - Chair Methodist Hospital, TX Christine Vande Velde, PhD University of , CAN Louis Kunkel, PhD - Chair Boston Children’s Hospital, MA Eric Wang, PhD University of Florida, FL Robert Baloh, MD, PhD Cedars-Sinai Medical Center, CA C. Chris Weihl, MD, PhD Washington University, MO Elisabeth Barton, PhD University of Florida, FL Noah Weisleder, PhD Ohio State University, OH Aaron Beedle, PhD SUNY-Binghamton, NY *R. Rodney Howell, MD University of Miami, FL Alan Beggs, PhD Boston Children’s Hospital, MA *Ex Officio Member 062017 Sanjay Bidichandani, MBBS, PhD University of Oklahoma, OK Stanley Appel, MD Methodist Hospital, TX Dean Burkin, PhD University of Nevada, NV Andrew Billin, PhD Gilead Sciences, CA Jeffrey Chamberlain, PhD University of Washington, WA Barry J. Byrne, MD, PhD University of Florida, FL M. Casey Childers, D.O., PhD University of Washington, WA Thomas Cheever, PhD NIAMS/NIH, MD Thomas Cooper, MD Baylor College of Medicine, TX Cristina Csimma, PharmD, MHP Csimma, LLC, MA Thomas Crawford, MD Johns Hopkins University, MD Kurt Fischbeck, MD NINDS/NIH, MD Merit Cudkowicz, MD, MSc Massachusetts General Hospital, MA Amelie Gubitz, PhD NINDS/NIH, MD John Day, MD, PhD Stanford University, CA John E. Howell Prometheus Partners Holdings, LLC, VA James Dowling, MD, PhD Hospital for Sick Children, CAN Louis Kunkel, PhD Boston Children’s Hospital, MA Fen-Biao Gao, PhD University of Massachusetts, MA Melanie Leitner, PhD Accelerating NeuroVentures, LLC, MA Matthew Harms, MD Columbia University, NY Elizabeth McNally, MD, PhD Northwestern University, IL Lawrence Hayward, MD, PhD University of Massachusetts, MA Jeffrey Rothstein, MD, PhD Johns Hopkins University, MD Michio Hirano, MD Columbia University, NY Lee Wrubel, MD EgonZehnder, NY Joe Kornegay, D.V.M., PhD Texas A & M University, TX Bruce Wentworth, PhD BMW Research Consulting, MA Clothilde Lagier-Tourenne, MD, PhD Massachusetts General Hospital, MA Dione T. Kobayashi, PhD AkeOla, MA Christian Lorson, PhD University of Missouri, MO Giovanni Manfredi, MD, PhD Weill Cornell Medical College, NY Nicholas Maragakis, MD Johns Hopkins University, MD Katherine Mathews, MD University of Iowa, IA Elizabeth McNally, MD, PhD Northwestern University, IL Lynn Megeney, PhD Health Research Institute, CAN MOVR ADVISORY BOARD Matthew Meriggioli, MD Rush University, IL

Daniel Miller, MD, PhD University of Washington, WA Barry Bryne, MD, PhD University of Florida Timothy Miller, MD, PhD Washington University, MO Elizabeth McNally, MD, PhD Northwestern University Carlos Moraes, PhD University of Miami, FL Gene Williams, MBA ProMIS Neurosciences Brett Morrison, MD, PhD Johns Hopkins University, MD James Berry, MD, MPH Massachusetts General Hospital Kanneboyina Nagaraju, DVM, PhD SUNY – Binghamton, NY John Day, MD, PhD Stanford University Bradley Olwin, PhD University of Colorado, CO Monkol Lek, PhD Yale University Udai Pandey, PhD University of Pittsburgh, PA Nicholas Maragakis, MD Johns Hopkins University John Ravits, MD University of California, San Diego, CA Sabrina Paganoni, MD, PhD Massachussetts General Hospital Louise R. Rodino-Klapac, PhD Ohio State University, OH Rich Finkel, MD Nemours Children's Health Center Alessandra Sacco, PhD Sanford Burnham Institute, CA R. Rodney Howell, MD University of Miami Rita Sattler, PhD Barrow Neurological Institute, AZ Michael Shy, MD University of Iowa, IA Melissa Spencer, PhD University of California, Los Angeles, CA Charlotte Sumner, MD Johns Hopkins University, MD

29 2018 ANNUAL REPORT 30 Financial FOR THE YEAR ENDED Report DECEMBER 31, 2018 (IN THOUSANDS) OTHER REVENUE CONTRIBUTIONS 3% 11% Assets Revenue Cash, cash equivalents, and investments $72,285 Special events, net $90,926 Receivables and other assets $10,735 Contributions $12,043 Fixed assets, net $645 Other Revenue $3,499 SPECIAL EVENTS, NET Total assets $83,665 Total revenue $106,468 86% Liabilities Accounts payable and accrued expenses $5,758 Research and training grants payable $10,469 GENERAL AND Line of credit $7,500 ADMINISTRATIVE Pension and postretirement plan $49,991 obligations 13% Total liabilities $73,718 Expenses Patient and community services $46,664 FUNDRAISING PATIENT AND Net Assets COMMUNITY Without donor restrictions $5,364 Research $18,763 14% SERVICES Professional public $12,220 With donor restrictions $4,583 health education 44% Total net assets $9,947 Fundraising $15,348 PROFESSIONAL General and administrative $13,714 PUBLIC HEALTH Total liabilities and net assets $83,665 Total expenses $106,709 EDUCATION 11% RESEARCH 18%

31 2018 ANNUAL REPORT 32 Recognition and Accreditations

ABOUT THE MUSCULAR DYSTROPHY ASSOCIATION MDA IS THE FIRST NONPROFIT MDA is committed to transforming the lives of people TO RECEIVE AN affected by muscular dystrophy, ALS, and related neuromuscular diseases. We do this through innovations in science and innovations in care. As the largest source of BBB TOP RATED LIFETIME MDA IS A GOLD-LEVEL funding for neuromuscular disease research outside of the ACCREDITED CHARITY ACHIEVEMENT GUIDESTAR PARTICIPANT CHARITY AWARD DEMONSTRATING federal government, MDA has committed more than by the American for “significant and ITS COMMITMENT TO $1 billion since our inception to accelerate the discovery Institute of lasting contributions to TRANSPARENCY Philanthropy the health and welfare of therapies and cures. Research we have supported is of humanity.” directly linked to approved, life-changing therapies across multiple neuromuscular diseases. We support the largest network of multidisciplinary clinics providing world-class care at more than 150 of the nation’s top medical institutions, and each year thousands of children and young adults learn vital life skills and gain independence at MDA Summer Camp and through recreational programs. MDA mda.org Muscular 800-572-1717 For more information, visit mda.org. Dystrophy [email protected] Association @MDAorg @MDAorg @MDAorg

33 2018 ANNUAL REPORT 34