2010 Annual Report

Make a Muscle. Make a Difference.® Message from the President & CEO

he cover of this report reflects a new branding of Among these were collaborations with federal TMDA, and in turn a new era in the Association’s agencies to accelerate research and build clini- development — an era in which a streamlining of cal trial infrastructure; and support for MDA’s our nationwide staff structure and plans for a trun- National Transitions Initiative, designed to help cated Labor Day Telethon have us poised for what people with childhood-onset muscle diseases we foresee as the most dynamic period of advance- who are now moving into adulthood thanks ment MDA has every experienced, in research and to decades of MDA support of clinical research service, and in the all-important fundraising effort and care. that enables them. Our new strategy has inspired the support of the greatest cadre of celebrities ever MDA’s “Make a Muscle, Make a Difference” to represent the Association, stars who’ve been campaign surged ahead in 2010 thanks “Making a Muscle and Making a Difference” for also to support from our national sponsors: MDA in top national media for the past year. Acosta, Bally Total Fitness, CITGO, Clear Channel Radio, ClubCorp, Dr Pepper Snapple As you’ll see in these pages, the increased aware- Group, ERA Franchise Systems, Harley- ness of our mission that they’ve spurred has helped Davidson Motor Company, the International achieve dramatic advances despite the effects on Association of Fire Fighters, Lowe’s, the the Association of our nation’s devastated economy. National Association of Letter Carriers, National The Association’s research effort forged ahead with Coalition of 7-Eleven Franchisees, Safeway, both basic and applied biomedical projects and saw and the Tall Cedars of Lebanon of North several promising treatments move from the labora- America. tory into clinical testing. MDA also launched the Muscle Walk, a Also in 2010, tens of thousands of children and new signature fundraising event that brings adults received health care services from neuromus- together MDA families, clinic teams, research- cular disease experts at some 200 MDA clinics and ers, camp counselors and sponsors. 38 MDA/ALS Centers, and nearly 3,500 children “Make a Muscle, Make a Difference” has attended 80 MDA summer camp sessions. meant that tough times are falling prey to a toughened Muscular Dystrophy Association, MDA expanded its use of online media in 2010, full of dedicated people and supported by providing families with myMuscleTeam, a care-coor- loyal sponsors and the public. dination tool, and with online educational seminars (“webinars”) on topics such as education, travel and With every best wish, medical care. At the same time, social media were integrated into MDA fundraising programs. MDA’s Advocacy Department supported several ini- tiatives in Washington, D.C., that have the potential Gerald C. Weinberg to improve the lives of those the Association serves.

The Make a Muscle, Make a Difference® campaign was created pro bono for MDA by E.B. Lane. 2 Report from the Chairman of the Board

hether you call it “bench to bedside,” Also in 2010, MDA awarded $1.4 million to W“laboratory to clinic,” or “microscope the biopharmaceutical company Repligen of to marketplace,” a major emphasis of MDA’s Waltham, Mass., to develop RG3039, an experi- research program in 2010 was clear: to move mental drug designed to raise levels of a protein promising treatments through the develop- that’s deficient in spinal muscular atrophy. ment pipeline — where possible, all the way to people with neuromuscular diseases. MDA funded a number of promising research projects at later stages along the development In amyotrophic lateral sclerosis, that’s meant pipeline while, at the same time, its basic science continuing MDA’s robust partnership with program continued to feed new ideas into its the ALS Therapy Development Institute beginning, ensuring a steady stream of therapeu- (ALS TDI) of Cambridge, Mass., where an tically focused projects. experimental compound that modifies the immune system’s behavior is a front-runner; Many of MDA’s “beginning of the pipeline” continued support of the five-center MDA grants in 2010 were to seasoned academic ALS Clinical Research Network; and funding researchers holding prestigious posts at major for Isis Pharmaceutials in Carlsbad, Calif., for universities. Others were to new entrants into the development of an innovative drug to block field, young people with recently minted doctor- toxic protein synthesis. ates in medicine and/or science who are now committed to MDA’s goals. In Duchenne and Becker muscular dys- trophies, the MDA DMD Clinical Research Most of the awards made to projects further Network began studies of heart dysfunction along the drug development pipeline have been and tests of two heart drugs, as well as stud- to biotechnology companies, uniquely positioned ies to fill in the gaps about the natural history to bring innovative drugs to market. of DMD in children younger than 3 and The downturn in the U.S. economy did not young men who are no longer walking. In change much in 2010, but neither did MDA’s addition, researchers gained crucial insights unwavering commitment to find treatments for into the immunologic barriers to gene the more than 40 diseases in the Association’s therapy in DMD/BMD. program. A drug called ataluren, developed by PTC Therapeutics of South Plainfield, N.J., with MDA support, showed promising results in R. Rodney Howell, M.D. boys with DMD or BMD caused by a certain type of genetic mutation.

3 Overview of 2010 Operations

Research

espite the prolonged recession in the U.S. and BMD. The network also began a study Deconomy, MDA research continued at a to compare the effectiveness of the cardiac rapid pace in 2010. The Association awarded drugs losartan and lisinopril in DMD. 90 grants for research in the diseases in its pro- gram, for a total of more than $33.1 million. Late in 2010, the network undertook two additional studies to develop outcome MDA researchers throughout the world contin- measures for DMD clinical trials in children ued to explore strategies they’ve been pursuing younger than 3 years old and in boys and and set out in several new directions as well. young men with DMD who are no longer walking. Most trials in DMD require partici- Translational research a major focus pants to be old enough to cooperate with for MDA strength tests and to be still walking. Being able to measure the effects of experimental MDA’s translational research program took treatments in participants who are not in center stage in 2010 in several disease catego- these categories will expand the reach of ries. (Translational research seeks to “translate” clinical trials in DMD. promising basic science into actual therapies.) Highlights of MDA’s translational research In October, results of a small safety achievements in 2010 follow. and feasibility trial of dystrophin gene therapy for DMD/BMD were announced DMD and BMD and showed that an unwanted immune response to the therapy occurred in some In 2010, several studies got under way under of the participants. In this trial, miniaturized the auspices of the MDA Duchenne Muscular dystrophin genes, encased in viral shells, Dystrophy (DMD) Clinical Research Network. were injected into the biceps muscles of The network consists of five U.S. centers that six participants. These results indicate that collaborate on clinical trials and studies designed immune system rejection of DMD/BMD to improve and standardize care for DMD and gene therapy is a factor that deserves major the related disease, Becker muscular dystrophy consideration as the field moves forward. (BMD). Also in October, New Jersey biotechnology In the fall, the network began studying the company PTC Therapeutics announced that natural history of heart function in DMD and the low-dose regimen of its experimental BMD, and its correlation to skeletal-muscle func- drug ataluren increased walking distance in tion and to specific mutations in the dystrophin boys with Duchenne muscular dystrophy gene. Mutations in this gene underlie both DMD (DMD) or Becker muscular dystrophy 4 “MDA is important for a variety of reasons, not the least of which is it funds — strategically and knowledgeably — good research.” John McCall Drug discovery consultant Chair of the spinal muscular atrophy development team National Institutes of Health

(BMD) who had a certain type of mutation mutations. Eleven of the 12 participants in this in the dystrophin gene. Ataluren, devel- trial produced the needed dystrophin protein oped with MDA support to PTC, makes after treatment with this drug, which is designed use of a strategy called stop codon read- to change the way cells read genetic instructions through. It’s designed to coax muscle cells for dystrophin. The drug is a synthetic molecule to ignore, or “read through,” molecular and makes use of a strategy called exon skip- stop signals in the dystrophin gene and ping. produce a functional dystrophin protein. The same month, biotechnology company AVI In April, the multinational pharmaceuti- BioPharma of Bothell, Wash., announced simi- cal company GlaxoSmithKline and the larly encouraging results for its exon-skipping Dutch biotechnology company Prosensa molecule, AVI-4658. MDA funded much of the announced encouraging results from an basic science research that made development early-stage trial of their experimental drug of exon-skipping drugs possible. PRO051/GSK2402968 in boys with DMD with specific types of dystrophin gene MDA has supported a strategy called myostatin inhibition for DMD, BMD and potentially other muscular dystrophies. Myostatin is a naturally occurring protein that limits muscle growth, and interfering with it could allow muscles to grow

5 larger and possibly stronger. In 2010, Acceleron ALS Pharma of Cambridge, Mass., began testing the myostatin inhibitor ACE-031 in people with MDA continued its partnership with the ALS DMD. Therapy Development Institute (ALS TDI) in Cambridge, Mass., giving the institution a Delaware biotechnology company Tivorsan new milestone-driven grant of $2.5 million Pharmaceuticals announced in September that in January, in addition to the $18 million it would develop an experimental drug for DMD previously awarded. ALS TDI is a nonprofit or BMD based on a protein called biglycan, research center focused exclusively on devel- which had been the subject of MDA-supported oping treatments for ALS. research. The goal of biglycan treatment is to improve the structure and function of the mus- In 2010, ALS TDI’s leading therapeutic cle-fiber membrane, which is fragile in DMD and candidate was ALS TDI 00846, an immune BMD because of a deficiency of dystrophin. system modulator.

Also in 2010, BioMarin Pharmaceutical of Also this year, MDA’s ALS Clinical Research Novato, Calif., began testing BMN195 in healthy Network, a consortium of five MDA/ volunteers. BMN195 is designed to increase pro- ALS centers located throughout the duction of utrophin, a protein that’s very similar United States, continued to forge ahead. to dystrophin and can probably compensate Established by MDA in 2008, each of the at least partially for the absence of dystrophin. five centers in the network receives support Utrophin research has been a major focus of from the Association to build infrastructure MDA’s basic science program for several years. and conduct clinical trials in ALS (amyo- trophic lateral sclerosis). In 2010, a trial to

6 compare the effects of a regular diet, a high-calorie diet and a high-calorie, high-fat diet in people with ALS continued.

A clinical trial of an experimental drug called ISIS-SOD1-Rx, developed by Isis Pharmaceuticals of Carlsbad, Calif., with MDA support, began in 2010. This is a drug to test a molecular strategy called antisense, to see whether it can block genetic instructions for a toxic protein Pompe disease called SOD1 in people with ALS who have mutations in the SOD1 gene. The clinical May 2010 saw the approval by the U.S. Food trial is designed to establish the safety pro- and Drug Administration of Lumizyme, a drug file of this new compound. specifically for late-onset Pompe disease (acid maltase enzyme deficiency). Biopharmaceutical Four other ALS clinical trials, while not company Genzyme of Cambridge, Mass., directly funded by MDA, were made pos- received approval from the FDA in 2006 to sible in large part by basic science research market the closely related drug Myozyme for conducted by MDA-supported investiga- infants and children with this metabolic muscle tors. In 2010, Neuraltus Pharmaceuticals of disorder. Myozyme and Lumizyme both replace Palo Alto, Calif., began testing its experi- the missing enzyme and were developed in mental drug NP001. This small-molecule part because of MDA-supported basic science drug, whose development is based on research on acid maltase. MDA research on the immune system in ALS, is designed to switch immune system SMA cells from an active, inflammatory mode to a more normal, healing mode in ALS. In December 2010, MDA awarded $1.4 million to the biopharmaceutical company Repligen of Sangamo BioSciences of Richmond, Calif., Waltham, Mass., for development of RG3039, an reported that its experimental compound experimental drug to treat spinal muscular atro- SB509 was safe and showed hints of possi- phy (SMA). RG3039 is designed to increase the ble efficacy in people with ALS. The devel- amount of fully functional SMN protein, which opment of SB509, designed to increase is deficient in people with SMA, by causing cells production of a protein called vascular to process the genetic instructions for SMN in a endothelial growth factor (VEGF), benefited different way. from MDA research on this molecule.

7 Health Care Services

he year 2010 saw enhancements to MDA’s MDA’s equipment program provided indi- Tprogram, as well as the continuation of core viduals with nearly 5,500 items of gently services. used durable medical equipment, from bath equipment and wheelchairs to hospi- Tens of thousands of individuals served by MDA tal beds and communication technology, received health care services from neuromuscular- donated by generous and caring individu- disease specialists at some 200 MDA clinics and 38 als. MDA also assisted with repairs to all MDA/ALS centers in the United States and Puerto types of medical equipment for those it Rico. Medical experts at MDA clinics conducted serves. medical exams and diagnostic consultations, and made referrals for assistive therapies to help main- In 2010, nearly 3,500 children ages 6-17 tain function, independence and quality of life. enjoyed a week of fun and friendship through nearly 80 MDA summer camp MDA clinics are at the forefront of research and sessions. More than 4,300 volunteers clinical care, and the Association is working on helped make this week extra special for many levels to ensure that its clinics continue to campers. meet the needs of those it serves. In 2010, MDA launched an online survey for families seen in MDA clinics called “Tell us about your MDA clinic,” as well as a Clinical Advisory Committee comprised of experts in neuromuscular care.

8 “MDA basically means support, and I think that’s clear enough for anybody to understand.” Benjamin F. Cumbo IV Graduate student, George Washington University Duchenne muscular dystrophy

MDA provided a wide range of support programs in 2010. Thousands of individu- als and families around the U.S. received help from facilitated MDA support groups, as well as from MDA-sponsored educational seminars; referral services; transitional services for youth with neu- romuscular disease who are entering adulthood; online chat sessions and MDA’s e-community, myMDA.

Two new support options got under way in 2010. MyMuscleTeam (which actually management, travel, higher education and more. debuted in December 2009) is an online The webinars are archived for viewing on mda.org. care coordination tool for families. MDA also launched MDA again partnered with Walgreens for its live expert-hosted webinars annual flu shot program, providing thousands on such topics as hous- of free inoculations to individuals served by the ing, accessibility, Association for whom a “simple case of the flu” research, medical could prove life-threatening.

9 Fall • October-December 2010 quest.mda.org

A dozen ways to ask for help

Hi-tech treadmills let nonwalkers ‘walk’

James Lupski’s Genetic Blueprint Tells the Tale of His CMT Professional and

Join a Muscle Public Health Information Walk!

DA takes its health information mission very Web pages logged more than 7.4 million page Mseriously, providing individuals and families views from visitors in 2010. Along with some affected by neuromuscular diseases with a wide 100 new projects completed during the year, variety of easy-to-understand information about MDA’s Online Services department continued neuromuscular diseases, current research and health to upgrade MDA’s Web infrastructure with care topics. MDA gears this information to the dif- more modern and robust technologies. fering needs of the individuals and families it serves, researchers, health care professionals, the news Television Production media and the public at large. In 2010, MDA’s Television Production Division MDA’s articulate and passionate spokespeople are completed a variety of projects for television, another important way MDA spreads the word radio and other media, including a satellite about the fight against neuromuscular diseases. media tour featuring MDA Chairman of the Board Rod Howell, which highlighted MDA’s Publications involvement in the eventual discovery of Myozyme to treat Pompe disease. Educational/ MDA’s award-winning Publications program informational videos were produced on a produced a steady stream of print and online number of topics for researchers, families information about research and health care, as and the general public, and public service well as articles about relevant legislation, valuable announcements featured a variety of celebrities services and inspiring individuals. In addition to the supporting MDA’s “Make a Muscle, Make a quarterly Quest magazine and bimonthly MDA/ALS Difference” multimedia campaign. Newsmagazine, the department posted an average of two online news articles a week in 2010, making MDA Ambassadors mda.org the “go-to” site for the latest and most accurate information about neuromuscular disease. Serving a third term as MDA National Goodwill MDA’s extensive free library of online publications Ambassador, 11-year-old Abbey Umali, of and articles was accessed by individuals affected by Redlands, Calif., crisscrossed the country with muscle diseases in more than 180 countries. her parents, speaking from the heart to groups, organizations and large audiences about MDA Online MDA’s quest to defeat muscle diseases. The self-assured and talented 11-year-old has a MDA’s award-winning website, mda.org, showcased form of Charcot-Marie-Tooth disease (CMT). the Association’s missions to the world, providing instant information about research, health care During the 2010 Telethon, Thomas Hale services, clinical trials and MDA programs. MDA’s Arrington III, of Chesapeake, Va., was named 10 “Every time I visit your site, I get good information about practically everything.” Comment left by a visitor to MDA’s website

the 2011 recipient of the Robert Ross MDA National Personal Achievement Award. Arrington, a clean-energy entrepreneur who promotes the use of renewable energy, has facioscapulohumeral muscular dystrophy (FSHD).

Luke Christie of Due West, S.C., served a sec- ond term as MDA National Youth Chairman. Luke, who has spinal muscular atrophy, is a former two-time MDA National Goodwill Ambassador (2006 and 2007) and in 2008 was named the first Harley-Davidson MDA Goodwill Ambassador. He turned 17 in 2010. Thomas Hale Arrington III, 2010 Personal Achievement Award recipient Augie and Lynne Nieto of Corona del Mar, Calif., served a fifth year as co-chairs of MDA’s The MDA Art Collection, which represents artists ALS Division. Augie, who turned 52 in 2010, with neuromuscular diseases from every state has lived with ALS since 2005. He continued and Puerto Rico, added six new pieces in 2010. to be “chief inspiration officer” despite the Selections from the more than 370 pieces in the progression of his disease, speaking via a Collection were the subject of gallery and museum communication device to organizations and shows across the country. the media about the need to cure ALS. MDA’s Augie’s Quest is spearheaded by the Nietos, Public Relations and has raised millions for MDA’s fast-track ALS initiative. As is evident on the front and back covers of this report, a cadre of celebrities flexed their biceps for Nancy O’Dell, co-anchor of the entertainment MDA’s “Make a Muscle, Make a Difference” public news show “Entertainment Tonight,” com- service advertising (PSA) campaign, including Reggie pleted her first full year as MDA National ALS Bush, Carrot Top, Don Francisco, Nigel Lythgoe, Ambassador. O’Dell, who also is a Telethon Kyle Massey, Natalie Morales, Blake Shelton, Alison co-host, spread awareness of MDA’s fight Sweeney, Triple H, Kurt Warner and Wynonna. PSAs against ALS through media interviews, public appeared in the Wall Street Journal, USA Today, appearances and public service announce- New York Post, and Time, Parade and Business ments. In June 2008, ALS claimed the life of Traveler magazines, as well as in thousands of TV, O’Dell’s mother. cable and radio spots. 11 Advocacy

DA’s Advocacy Department works year- Among the many health policy initiatives Mround to keep the needs of the neuromus- supported by MDA in 2010 were: cular disease community in the minds of the Washington, D.C., policymakers who craft legis- • National Transitions Initiative for MDA lation, research priorities and health policy. The community members with pediatric MDA Fly Out — which occurs during the con- muscle diseases who are transitioning gressional August recess in the representatives’ into adulthood home districts — is just one of the innovative, cost-effective ways MDA enabled the maximum • Collaborations with federal agencies number of individuals and families affected by and voluntary health organizations to muscle diseases to make their concerns and pri- accelerate research, build critical clinical orities known to their legislators in 2010. trial infrastructure, and expand out- reach and awareness among medical MDA’s National Task Force on Public Awareness and patient communities provided insight and guidance to the Association on policy issues and activities of importance to • SMA Treatment Acceleration Act of people with disabilities. The 12 professional and 2009 community leaders on the Task Force all are affected by neuromuscular diseases, and come • Medicare Competitive Bidding Repeal from such fields as law, education, homeland Act security, computer technology, engineering and communications. • Achieving a Better Life Experience (ABLE) Act

It’s through your outstanding work that families and individuals with neuromuscular disease like me are living rewarding, productive lives fully integrated into their communities. Christopher Rosa Director, Office of Special Services for Students with Disabilities Queens College of the City University of New York Becker muscular dystrophy

12 Management and Fundraising

or MDA, 2010 was the “Year of Muscle.” October that the Telethon will move to a shorter FAll our national sponsors rallied around (six-hour) prime-time format in 2011. MDA’s “Make a Muscle, Make a Difference” campaign. A new MDA signature event was After 45 years of dedicated hosting of MDA’s launched in 2010: the MDA Muscle Walk. signature event, Jerry Lewis Billed as MDA’s “local family reunion,” it announced his retirement brings together individuals and families served as Telethon host, saying “It’s by MDA, clinic teams, researchers, camp time for an all new Telethon counselors and MDA sponsors to raise money era,” but also vowing, “I’ll and participate in a fun, accessible walk. MDA never desert MDA and my also integrated social media into Lock-Up and kids.” Muscle Walk fundraising strategies so that Jailbirds and Muscle Walk participants could In late 2010, MDA dedicated a use Facebook and Twitter to drive donations new 15,000-square-foot build- to MDA. ing at its national headquarters in Tucson: the Rachel Ann The Association began accepting donations by Perkinson Center. The result of text message, attracting more than $50,000 in a $6 million bequest from the new MDA Labor Day Telethon income from Perkinson estate, the acces- people using their cell phones to text “MDA” sible, environmentally friendly to 20222. building houses MDA’s research and health care The 2010 MDA Telethon featured wall-to-wall services programs, and pro- entertainment, information about research, vides much-needed space and profiles of individuals living with muscle for meetings and training. diseases, raising $58,919,838 for MDA’s mis- sions of help and hope. MDA announced in

“The thing that MDA has done for me that I am most grateful for is they have given me the power to DO something. If we all do something, I know that we will see the finish line in our battle!” Susan Hanna Muscle Walk participant Mother of a son with Duchenne muscular dystrophy 13 Neuromuscular Diseases Included in MDA’s Programs Usual Age Group/Type of Onset Disease Characteristics

Muscular Dystrophies Weakness of all muscle groups accompanied by delayed Birth to relaxation of muscles after contraction. Affects face, feet, hands Myotonic adulthood and neck first. Progression is slow, sometimes spanning 50 to 60 years. General muscle weakness and wasting, affecting pelvis, upper arms and upper legs first. Duchenne progresses slowly, yet 2 to 6 years Duchenne eventually involves all voluntary muscles. Survival is rare beyond the late 20s. Symptoms almost identical to Duchenne yet less severe. Affects pelvis, upper arms and upper legs first. Becker pro- 2 to 16 years Becker gresses more slowly than Duchenne and survival runs well into middle age.

Weakness and wasting, affecting shoulder girdle and pelvic Late childhood girdle first. Disease usually progresses slowly. Variable cardio- Limb-Girdle to middle age pulmonary complications may occur in later stages.

Facial muscle weakness, with weakness and wasting of the Childhood to shoulders and upper arms. Progressing slowly with some peri- Facioscapulohumeral early adulthood ods of rapid deterioration, disease may span many decades.

Generalized muscle weakness, with possible joint contractures Congenital At birth or infancy resulting from shortening of muscles. Disease progresses very slowly. Weakness is variable.

First affects muscles of eyelids and throat. While progression is Early adulthood slow, weakening of throat muscles in time causes swallowing Oculopharyngeal to middle age difficulties.

Early adulthood Weakness and wasting of muscles of the hands, forearms and Distal to middle age lower legs. Progresses slowly and is rarely life-threatening.

Weakness and wasting of shoulder, upper arm and shin mus- Childhood to cles. Joint contractures are common. Disease progresses slowly, Emery-Dreifuss early teens with cardiac complications common. Motor Neuron Diseases Progressive wasting and weakness of all voluntary muscles, Amyotrophic Lateral with cramps and muscle twitches common. ALS first affects Adulthood Sclerosis (ALS) legs, arms and/or throat muscles. Survival rarely exceeds five years after onset, without respiratory intervention.

Infantile Progressive Spinal Generalized muscle weakness, weak cry, trouble swallowing Birth to 3 months and sucking, and breathing distress. Life span rarely exceeds Muscular Atrophy age 2.

14 Usual Age Group/Type of Onset Disease Characteristics

Weakness in arms, legs, upper and lower torso, often with Intermediate Spinal 6 months skeletal deformities. Disease progresses rapidly. Respiratory to 3 years Muscular Atrophy problems can shorten life.

Juvenile Spinal Weakness in leg, hip, shoulder, arm and respiratory muscles. 1 to 15 years Muscular Atrophy Disease progresses slowly. Life span is unaffected.

Adult Spinal Generalized muscle weakness with muscle twitches common. 18 to 50 years Muscular Atrophy Disease progression varies.

Spinal-Bulbar Weakness of limb muscles, especially legs, and of muscles 15 to 60 years involved in talking, chewing and swallowing. Occurs in men. Muscular Atrophy Slowly progressive over decades. Inflammatory Myopathies

Weakness of neck and limb muscles, sometimes with pain. Childhood to Disease severity and progression vary by individual. Sometimes Polymyositis late adulthood associated with malignancy. Often responds to drug therapy.

Weakness of neck and limb muscles, often with pain. Skin rash Childhood to typically affects cheeks, eyelids, neck, chest and limbs. Disease Dermatomyositis late adulthood severity and progression vary. Sometimes associated with malignancy. Often responds to drug therapy.

Weakness of arms, legs and hands, especially thighs, wrists Inclusion-Body Myositis After age 50 and fingers. Sometimes involves swallowing muscles. Slowly progressive. More common in men than women. Diseases of the Neuromuscular Junction

Weakness and fatigability of muscles of the eyes, face, neck, Childhood to throat, limbs and/or trunk. Weakness may fluctuate. Disease Myasthenia Gravis adulthood progression varies. Drug therapy and/or removal of thymus gland often effective.

Weakness and fatigue of hip and leg muscles with aching back Lambert-Eaton and thigh muscles common. Lung tumor is often present. Adulthood Myasthemic Syndrome Progression varies with success of drug therapy and treatment of any malignancy.

Congenital Myasthenic Infancy or Generalized weakness and fatigability of voluntary muscles, childhood, including those controlling eye movement, swallowing and Syndromes can be later breathing. Varies in severity, and weakness can fluctuate.

15 Neuromuscular Diseases Included in MDA’s Programs Usual Age Group/Type of Onset Disease Characteristics

Diseases of the Peripheral Nerves Weakness and atrophy of muscles of hands and lower legs, Charcot-Marie-Tooth Childhood to young with foot deformities and some loss of sensation in feet. adulthood Disease Disease progression usually slow.

Impairment of balance and limb coordination, muscle weak- Childhood to ness and loss of sensation. Severity and progression of disorder Friedreich's Ataxia adolescence vary. Often associated with diabetes and heart disease.

Delayed development of motor skills. Muscle weakness affects Dejerine-Sottas Infancy to early hands and legs and may involve impairment of sensation. childhood Disease Severity and progression of disease vary. Metabolic Diseases of Muscle

Muscle cramps often occur after exercise. Intense exercise can Phosphorylase Childhood to cause muscle destruction and possible damage to kidneys. adolescence Deficiency Reducing strenuous exercise can lessen severity.

For infants, disease is generalized and severe with heart, Acid Maltase Infancy to liver and tongue enlargement common. Adult form involves Deficiency adulthood weakness of upper arms, legs, trunk and respiratory muscles. Progression varies. Muscle fatigue which upon exercise can lead to severe cramps, Phosphofructokinase Childhood to nausea, vomiting, muscle damage and discoloration of urine. adulthood Deficiency Progression varies.

Generalized weakness and muscle wasting. Heart involvement Debrancher Early childhood and enlarged liver may occur with infantile form. Episodes of Enzyme Deficiency to adulthood low blood sugar are common. Variable progression. Muscle symptoms may be delayed until teens or adulthood.

Generalized muscle weakness with droopy eyelids and inability Early childhood to walk. Brain is often involved, with seizures, deafness, loss of Mitochondrial Myopathy to adulthood balance and vision, and retardation common. Progression and severity vary widely.

Varied weakness of shoulder, hip, face and neck muscles. Early childhood Carnitine Deficiency Progression varies and carnitine supplementation can be effective.

Inability to sustain moderate prolonged exercise. Prolonged Carnitine Palmityl Young exercise and/or fasting can cause severe muscle destruction adulthood Transferase Deficiency with urine discoloration and kidney damage. Severity varies.

Phosphoglycerate Childhood to Muscle pain and weakness, with muscle damage and urine dis- Kinase Deficiency adolescence coloration possible after vigorous exercise. Severity varies.

16 Usual Age Group/Type of Onset Disease Characteristics Muscular pain, cramps, muscle damage and urine Phosphoglycerate Childhood to discoloration possible following intense exercise of brief dura- adulthood Mutase Deficiency tion. Severity varies.

Intolerance of intense exercise with muscle damage and urine Lactate Dehydrogenase Childhood to discoloration possible following strenuous physical activity. adolescence Deficiency Severity of disorder varies.

Muscle fatigue and weakness during and after exertion, with Myoadenylate Deaminase Early adulthood muscle soreness or cramping. Condition is nonprogressive and to middle age Deficiency severity varies.

Myopathies Due to Endocrine Abnormalities Weakness in upper arm and upper leg muscles with some Childhood to evidence of wasting. Usually improves with treatment of under- Hyperthyroid Myopathy adulthood lying thyroid condition.

Weakness of arm and leg muscles. Stiffness, muscle pain and Childhood to cramps common. Usually improves with treatment of underly- Hypothyroid Myopathy adulthood ing thyroid condition. Other Myopathies

Muscle stiffness and cramps after periods of rest. Condition Early childhood Myotonia Congenita causes discomfort but is not life-threatening.

Poor or difficult relaxation of muscles, which may worsen after Paramyotonia Childhood to exposure to cold or exercise. Often associated with hyperka- Congenita early adulthood lemic periodic paralysis. Condition causes discomfort but isn’t life-threatening.

Infancy to Delayed motor development. Hip displacement is not uncom- Central Core Disease childhood mon. Condition can be stable to slowly progressive.

Delayed motor development. Weakness of arm, leg, trunk, face Infancy to and throat muscles. Respiratory involvement common. Severity Nemaline Myopathy childhood and progression vary.

Drooping of upper eyelids, facial weakness. Weakness of the limbs and trunk muscles. Patients almost always have no Infancy Myotubular Myopathy reflexes. Respiratory involvement is possible. Disease pro- gresses slowly.

Periodic Paralysis Episodes of generalized muscle weakness. Hyperkalemic type Childhood to may be associated with paramyotonia congenita. Frequency of — Hypokalemic adulthood — Hyperkalemic attacks and severity vary. May respond to drug therapy.

17 MDA-Supported Researchers (For calendar year 2010)

Arizona Orange District of Columbia Phoenix University of California, Irvine Washington St. Joseph’s Hospital & Medical Center Virginia Kimonis, M.D., M.R.C.P. Children’s Research Institute (CNMC) Fu-Dong Shi, M.D., Ph.D. Palo Alto Eric Hoffman, Ph.D. Terence Partridge, Ph.D. Tempe Palo Alto Institute for Research & Christopher Spurney, M.D. Arizona State University Education N. Jeanne Wilson-Rawls, Ph.D. Thomas Rando, M.D., Ph.D. Florida Tucson Redwood City Gainesville University of Arizona Glialogix University of Florida Ronald Allen, Ph.D. Mark Moore, Ph.D. Sean Forbes, Ph.D. Daniela Zarnescu, Ph.D. Sacramento Steven Lipshultz, M.D. California University of California, Davis Miami Craig McDonald, M.D. University of Miami Davis Lisa Baumbach, Ph.D. University of California San Francisco Michael Benatar, Ph.D. Gino Cortopassi, Ph.D. California Pacific Medical Center Gavriel David, Ph.D., M.D. Michael Ferns, Ph.D. Robert Miller, M.D. Flavia Fontanesi, Ph.D. David Richman, M.D. Connie Yamashita Carlos Moraes, Ph.D. La Jolla University of California Georgia The Scripps Research Institute Douglas Gould, Ph.D. Pritilekha Deka, Ph.D. Atlanta Santa Cruz Emory University Matthew Disney, Ph.D. University of California Ya Wen Liu, Ph.D. Luciano Apponi, Ph.D. Manuel Ares, Ph.D. Gary Bassell, Ph.D. University of California South San Francisco Tamara Caspary, Ph.D. Ju Chen, Ph.D. iPierian Jonathan Glass, M.D. Albert La Spada, M.D., Ph.D. John Dimos, Ph.D. Madhuri Hegde, Ph.D. G. Diane Shelton, D.V.M., Ph.D. Wilfried Rossoll, Ph.D. Stanford Los Angeles Stanford University Augusta Cedars-Sinai Medical Center Michele Calos, Ph.D. Medical College of Georgia Research Ronald Victor, M.D. Lorene Nelson, Ph.D. Institute Lin Mei, M.D., Ph.D. University of California, Los Angeles Colorado Linda Baum, M.D., Ph.D. Illinois Carmen Bertoni, Ph.D. Aurora Chicago Bennett Novitch, Ph.D. University of Colorado, Denver University of Chicago James Tidball, Ph.D. Anshutz Medical Campus & Downtown Campus Elizabeth McNally, M.D., Ph.D. University of Southern California Kurt Beam, Ph.D. Kamal Sharma, Ph.D. Valerie Askanas, M.D., Ph.D. William Betz, Ph.D. University of Illinois Chien-Ping Ko, Ph.D. Connecticut Jesus Garcia-Martinez, M.D., Ph.D. Merced New Haven Matthew Meriggioli, M.D. University of California Yale University JianRong Sheng, Ph.D. Wei-Chun Chin, Ph.D. Lynn Cooley, Ph.D. Antonio Giraldez, Ph.D. 18 Maywood Maryland Massachusetts General Hospital Loyola University Chicago Stritch Baltimore Nazem Atassi, M.D. School of Medicine Johns Hopkins University Merit Cudkowicz, M.D. Renzhi Han, Ph.D. Mohamed Farah, Ph.D. Anne-Marie Wills, M.D. Indiana Se-Jin Lee, M.D., Ph.D. Tufts University School of Medicine Indianapolis Brett Morrison, M.D., Ph.D. Yongjie Yang, Ph.D. Indiana University Jeffrey Rothstein, M.D., Ph.D. Shanthini Sockanathan, Ph.D. Cambridge Tatiana Foroud, Ph.D. Harvard College William Groh, M.D. University of Maryland Alexander Schier, Ph.D. West Lafayette Robert Bloch, Ph.D. Diana Ford, Ph.D. Everett Purdue University 4S3 BIOSCIENCE Shihuan Kuang, Ph.D. Rockville Dustin Armstrong, Ph.D. Iowa Food and Drug Administration Jakob Reiser, Ph.D. Waltham Iowa City Repligen Corporation University of Iowa Massachusetts James Rusche, Ph.D. Kevin Campbell, Ph.D. Boston Watertown Kentucky Beth Israel Deaconess Medical Center Boston Biomedical Research Institute Zolt Arany, M.D., Ph.D. Lexington Jeffrey Miller, Ph.D. University of Kentucky Research Brigham and Women’s Hospital Inc. Worcester Foundation Pavel Ivanov, Ph.D. University of Massachusetts Medical Stefan Stamm, Ph.D. Calum MacRae, M.B., Ch.B, Ph.D. School Mohammad Salajegheh, M.D. Michael Francis, Ph.D. Louisville Xin Wang, Ph.D. University of Louisville Research Michigan Foundation Children’s Hospital — Boston Ann Arbor Ashok Kumar, Ph.D. Basil Darras, M.D. Steven Greenberg, M.D. University of Michigan Maine Louis M. Kunkel, Ph.D. John Kim, Ph.D. Bar Harbor The Jackson Laboratory Harvard Medical School Laurent Bogdanik, Ph.D. Marcia Haigis, Ph.D. Andrew Lassar, Ph.D.

19 Rochester University of Rochester Robert Griggs, M.D. Masayuki Nakamori, M.D., Ph.D. North Carolina Chapel Hill University of North Carolina Joe Kornegay, D.V.M., Ph.D. Charlotte Carolinas Medical Center Susan Sparks, M.D., Ph.D. Durham Duke University Medical Center Paul Rosenberg, M.D. Detroit New York Winston-Salem Wayne State University Albany Wake Forest University Health John Kamholz, Ph.D. State University of New York Sciences Michael Shy, M.D. Li Niu, Ph.D. Osvaldo Delbono, M.D., Ph.D. Minnesota Bronx Ohio Minneapolis Albert Einstein College of Medicine of Cincinnati University of Minnesota, Twin Cities Yeshiva University University of Cincinnati Atsushi Asakura, Ph.D. Jennifer Aguilan, Ph.D. Rhett Kovall, Ph.D. John Day, M.D, Ph.D. Chi-Wing Chow, Ph.D. Tom Thompson, Ph.D. James Ervasti, Ph.D. New York Dawn Lowe, Ph.D. Cleveland Columbia University Cleveland Clinic Foundation Rochester Hasan Akman, Ph.D. Andrea Ladd, Ph.D. Mayo Clinic Oliver Hobert, Ph.D. Lan Zhou, M.D., Ph.D. Michael Barry, Ph.D. Columbia University in the City of Andrew Engel, M.D. Columbus New York, Morningside Bruce Horazdovsky, Ph.D. Ohio State University Patricia Richard, Ph.D. Arthur Burghes, Ph.D. Missouri Columbia University College of Jill Rafael-Fortney, Ph.D. Columbia Physicians and Surgeons Research Institute at Nationwide University of Missouri Howard J. Worman, M.D. Children’s Hospital Dawn Cornelison, Ph.D. Kevin Flanigan, M.D. Dongsheng Duan, Ph.D. Columbia University Medical Center Jerry Mendell, M.D. Christian Lorson, Ph.D. Veronica Hinton, Ph.D. Michio Hirano, M.D. Christopher Pierson, M.D., Ph.D. St. Louis Hiroshi Mitsumoto, M.D. Pennsylvania Washington University Umrao Monani, Ph.D. Philadelphia Robert Baloh, M.D., Ph.D. Livio Pellizzoni, Ph.D. Anne Connolly, M.D. Children’s Hospital of Philadelphia Eric Schon, Ph.D. David Lynch, M.D., Ph.D. Marc Diamond, M.D. Ji-Yeon Shin, Ph.D. Paul Golumbek, M.D., Ph.D. Thomas Jefferson University Didier Hodzic, Ph.D. The Hospital for Special Surgery Ya-Ming Hou, Ph.D. Jeffrey Milbrandt, M.D., Ph.D. Dale Lange, M.D. Dena Jacob, Ph.D. Timothy Miller, M.D., Ph.D. Memorial Sloan-Kettering Cancer University of Pennsylvania Jindrich Soltys, Ph.D., D.V.M. Center Conrad Weihl, M.D., Ph.D. Nancy M. Bonini, Ph.D. Mary Baylies, Ph.D. Clara Franzini-Armstrong, Ph.D. New Mexico New York University Michael Granato, Ph.D. Albuquerque James Salzer, M.D., Ph.D. Todd Lamitina, Ph.D. University of New Mexico Weill Cornell Medical College Tennessee Richard Cripps, D. Phil. Giovanni Manfredi, M.D., Ph.D. Memphis St. Jude Children’s Research Hospital Brian Freibaum, Ph.D. 20 Nashville Utah Australia Vanderbilt University Salt Lake City Clayton Jun Li, M.D., Ph.D. Sfida BioLogic Inc. Monash University Allison Limpert, Ph.D. John Manfredi, Ph.D. Christina Mitchell, Ph.D. Texas University of Utah Concord Dallas Russell Butterfield, M.D., Ph.D. Anzac Health & Medical Research UT Southwestern Medical Center Gabrielle Kardon, Ph.D. Foundation Steve Cannon, M.D., Ph.D. Kathryn Swoboda, M.D. Garth Nicholson, M.D., Ph.D. Ronald Haller, M.D. Virginia Melbourne Galveston Charlottesville University of Melbourne University of Texas Medical Branch University of Virginia Gordon Lynch, Ph.D. Premkumar Christadoss, M.B.B.S. Mani Mahadevan, M.D. Parkville Houston Ramesh Yadava, Ph.D. Murdoch Childrens Research Institute Baylor College of Medicine Falls Church David Thorburn, Ph.D. Thomas Cooper, M.D. Lewin Group Perth Maria de Haro, Ph.D. Paul Hogan University of Western Australia Susan Hamilton, Ph.D. Steve Wilton, Ph.D. Lee-Jun Wong, Ph.D. Washington Seattle Belgium Methodist Neurological Institute Fred Hutchinson Cancer Research Stanley H. Appel, M.D. Antwerpen Center Flanders Institute for Biotechnology University of Texas Health Science Zejing Wang, M.D., Ph.D. Albena Jordanova, Ph.D. Center University of Washington Vihang Narkar, Ph.D. Canada Glen Banks, Ph.D. Irina Serysheva, Ph.D. Montreal Joel Chamberlain, Ph.D. Centre Hospitalier de l’Universite de San Antonio Stephen Hauschka, Ph.D. Montreal (CHUM) Research Center University of Texas Health Science Guy Odom, Ph.D. Guy Rouleau, M.D., Ph.D. Center Leo Pallanck, Ph.D. Holly Van Remmen, Ph.D. Zipora Yablonka-Reuveni, Ph.D. CRCHUM — Hopital Notre-Dame Bernard Brais, M.D.

21 Nijmegen Radboud University Nijmegen Medical Centre Berend Wieringa, Ph.D. Singapore National University of Singapore Reshma Taneja, Ph.D. Switzerland Geneva Université de Genève Frederique Ruf-Zamojski, Ph.D. United Kingdom London Institute of Child Health, UCL Francesco Muntoni, M.D. Institut de Recherches Cliniques de Greece Institute of Neurology, UCL Montréal Athens Henry Houlden, M.D., Ph.D., M.R.C.P. Artur Kania, Ph.D. Hellenic Pasteur Institute Oswestry McGill University Socrates Tzartos, Ph.D. Keele University Heather Durham, Ph.D. Israel Glenn Morris, D. Phil. Josephine Nalbantoglu, Ph.D. Petha Tikva Oxford Ottawa Tel Aviv University University of Oxford Ottawa Hospital Research Institute Daniel Offen, Ph.D. Kay Davies, M.A., Ph.D. Rashmi Kothary, Ph.D. Raanana Lynn Megeney, Ph.D. Open University of Israel Michael Rudnicki, Ph.D. Miriam Souroujon, Ph.D. Luc Sabourin, Ph.D. Rehovot University of Ottawa Weizmann Institute of Science Bernard Jasmin, Ph.D. Ben-Zion Shilo, Ph.D. Aymeric Ravel-Chapuis, Ph.D. Ilona Skerjanc, Ph.D. Italy Quebec Genova Université Laval Fondazione Istituto Italiano di Jean-Pierre Julien, Ph.D. Tecnologia Maria Pennuto, Ph.D. Chile Milan Santiago Fondazione Centro San Raffaele del Pontificia Universidad Catolica de Chile Monte Tabor [San Raffaele Scientific Enrique Brandan, Ph.D. Insitute] France Davide Gabellini, Ph.D. Paris Rome Universite de Montreal Research Provincia Italiana CFIC-Istituto Centre (Notre-Dame Hospital) Dermopatico dell’ Immacolata Edor Kabashi, Ph.D. Carlo Gaetano, M.D. Germany Netherlands Hannover Leiden Medizinische Hochschule Hannover LUMC (MHH) Silvere van der Maarel, Ph.D. Christoph Fahlke, M.D.

22 Directory of MDA Clinics

(As of July 2011) (*Directors of MDA/ALS Centers)

Alabama Little Rock Sacramento Birmingham University of Arkansas for Medical University of California, Davis Medical The Children’s Hospital Sciences Center Director: Director: Co-Directors: Charles R. Law, M.D. Stacy Rudnicki, M.D.* Craig McDonald, M.D. Jay J. Han, M.D. University of Alabama at Birmingham California Hospital Downey San Bernardino Co-Directors: Rancho Los Amigos National Loma Linda University Gwendolyn C. Claussen, M.D. Rehabilitation Center Director: Shin J. Oh, M.D. Director: David J. Michelson, M.D. Luis Antonio Montes, M.D. Alaska San Diego Children’s Hospital Anchorage Fresno Director: Alaska Neurology Center Community Medical Centers Carla M. Grossman, M.D. Director: Director: Franklin Ellenson, M.D. Jeffrey Rosenfeld, M.D. San Francisco California Pacific Medical Center Arizona Los Angeles Hospital of the Good Samaritan/USC Co-Directors: Phoenix Co-Directors: Robert G. Miller, M.D.* St. Joseph’s Hospital and Medical Valerie Askanas, M.D., Ph.D. Jonathan Katz, M.D. * Center W. King Engel, M.D.* Co-Directors: UCSF Medical Center John Bodensteiner, M.D. University of California, Los Angeles Cathy Lomen-Hoerth, M.D.* Shafeeq Ladha, M.D.* Co-Directors: UCSF Medical Center/UCSF Children’s Michael C. Graves M.D.* Tucson Hospital Perry B. Shieh M.D., Ph.D. Children’s Clinics for Rehabilitative Director: Services Madera Jonathan B. Strober, M.D. Director: Children’s Hospital Central CA Stanford Timothy Miller, M.D. Pediatric Neurology Stanford University Director: The Mucio F. Delgado Clinic for Co-Directors: Raymund Fernandez, M.D. Neuromuscular Disorders Yuen Tat So, M.D. Director: Orange Ching H. Wang, M.D., Ph.D. Katalin Scherer, M.D.* Children’s Hospital of Orange County Sylmar Director: University of Arizona Health Sciences Olive View — UCLA Medical Center Samuel R. Rosenfeld, M.D. Center Director: Director: University of California, Irvine Medical Shri K. Mishra, M.D., M.S. Lawrence Z. Stern, M.D. Center Colorado Co-Directors: Arkansas Aurora Tahseen Mozaffar, M.D.* University of Colorado Fort Smith Annabel Kim Wang, M.D. Cooper Clinic Director: Director: Steven P. Ringel, M.D. * William A. Knubley, M.D.

23 Tampa St. Joseph’s Children’s Hospital of Tampa Director: Raymond J. Fernandez, M.D. Georgia Atlanta Children’s Healthcare of Atlanta at Scottish Rite Co-Directors: Edward Goldstein, M.D. Joanne Janas, M.D. Emory University Co-Directors: Jonathan D. Glass, M.D.* Taylor B. Harrison, M.D. The Children’s Hospital Florida Augusta Director: Gainesville Medical College of Georgia Health Dennis J. Matthews, M.D. Shands Hospital Director: Connecticut Director: Jerry Ned Pruitt II, M.D. S.H. Subramony, M.D. New Britain Dalton Hospital for Special Care Jacksonville MDA Emory Clinic at Dalton, GA Co-Directors: Mayo Clinic — Jacksonville Director: Jinsy Andrews, M.D.* Director: Linton Cooke Hopkins, M.D. Kevin J. Felice, D.O. Kevin B. Boylan, M.D. Charles H. Whittaker, M.D. Hawaii Nemours Children’s Clinic at Kailua New Haven Jacksonville Castle Medical Center Yale University Director: Co-Directors: Co-Directors: David N. Hammond, M.D. Kevin G. Kimata, M.D. Jonathan M. Goldstein, M.D.* Jupiter Jayson H. Takata, M.D. Geoffrey Miller, M.D. Jupiter Medical Center Brain Smith, M.D. Idaho Co-Directors: Boise Delaware Linda Pao, M.D. Idaho Elks Rehabilitation Hospital J. Mark Stafford, D.O. Wilmington Director: Alfred I. duPont Hospital for Children Miami Robert H. Friedman, M.D. Co-Directors: University of Miami School of Illinois Laura S. Inselman, M.D. Medicine William G. Mackenzie, M.D. Co-Directors: Chicago Mena Scavina, D.O. Michael Benatar, M.D. Northwestern Memorial Faculty Foundation Christiana Care Health Services Khema Ram Sharm, M.D. Director: Co-Directors: Ashok Verma, M.D, D.M. * Robert Sufit, M.D. Charles Bean, M.D. Orlando Enrica Arnaudo, M.D., Ph.D. Orlando Regional Lucerne Hospital Shriners Hospital for Children — District of Columbia Director: Chicago Hospital Michael J. Creamer, D.O. Director: Children’s National Medical Center Kenneth Silver, M.D. Co-Directors: Sarasota Olga M. Morozova, M.D. Sarasota Memorial Hospital’s Institute University of Illinois, Chicago Ana Carolina Tesi Rocha, M.D. for Advanced Medicine Director: Julie Rowin, M.D.* Georgetown University Hospital Director: Director: Gregory P. Hanes, M.D. Peoria Michael Sirdofsky, M.D. St. Petersburg OSF St. Francis Medical Group Center St. Anthony’s Neurology Group Co-Directors: Director: Greg Blume, M.D. Allan S. Weiss, M.D. Christopher M. Zallek, M.D. 24 Springfield Kentucky Maryland Koke Mill Medical Center Louisville Baltimore Co-Directors: Baptist Hospital East Johns Hopkins University Michael R. Pranzatelli, M.D. Director: Co-Directors: Zeng Wang, M.D., Ph.D. Gregory L. Pittman, M.D. Thomas O. Crawford, M.D. Daniel Drachman, M.D. Urbana Louisiana Carle Physician Group Jeffrey D. Rothstein, M.D., Ph.D.* Alexandria Director: Rapides Regional Medical Center Massachusetts Robert E. Cranston, M.D. Director: Boston Indiana Charles Ugokwe, M.D., M.P.H.T.M. Brigham & Women’s Hospital Director: Elkhart Baton Rouge Anthony A. Amato, M.D. Elkhart Clinic LLC The Baton Rouge Clinic, AMC Director: Director: Children’s Hospital Boston Thomas Vidic, M.D. Barbara Jean Golden, M.D. Co-Directors: Basil Darras, M.D. Evansville The NeuroMedical Center Fred Shapiro, M.D. St. Mary’s Medical Center Director: Director: Carolyn C. Baker, M.D. Massachusetts General Hospital Raymond Nicholson Jr., M.D. Co-Directors: Lafayette Merit E. Cudkowicz, M.D.* Fort Wayne Our Lady of Lourdes Regional Medical Brian Tseng, M.D., Ph.D. Lutheran Hospital Center Co-Directors: Director: TUFTS Medical Center Hospital Jerry L. Mackel, M.D. Wael A. Karim, M.D., A.P.M.C. Director: C.J. Ottinger, M.D. Harry C. Webster, M.D. Monroe Indianapolis St. Francis Community Health Center Burlington Indiana University Hospital Director: Lahey Clinic Foundation Co-Directors: Aristoteles Pena-Miches, M.D. Director: Cynthia L. Bodkin, M.D. Jayashri Srinivasan M.D., Ph.D., New Orleans Shashank J. Davé, D.O. M.R.C.P. (UK) Robert M. Pascuzzi, M.D. Children’s Hospital Co-Directors: Springfield Methodist Hospital of Indiana Maria Weimer, M.D. Baystate Medical Center Director: Ann Henderson Tilton, M.D. Director: Laurence E. Walsh, M.D. Joaquin Wong, M.D. George Baquis, M.D. Iowa Louisiana State University Worcester Des Moines Director: UMass Memorial Medical Center Iowa Health Des Moines Amparo Gutierrez, M.D. Director: Director: Robert H. Brown, M.D., D.Phil.* Shreveport Calvin Jay Hansen, M.D. Louisiana State University Health Michigan Iowa City Science Center, Shreveport Ann Arbor University of Iowa Hospitals & Clinics Director: University of Michigan Director: Robert Schwendimann, M.D. Co-Directors: Katherine Mathews, M.D. Maine James Dowling, M.D., Ph.D. Joseph Hornyak, M.D., Ph.D. Kansas Bangor Kansas City Eastern Maine Medical Center Detroit University of Kansas Medical Center Director: Children’s Hospital of Michigan Director: Peter Keebler, M.D. Director: Arthur Dick, M.D.* Huiyuan Jiang, M.D., Ph.D. Portland Wichita Maine Medical Center WSU Physician Group Adult MDA Via Christi Medical Center Director: Clinic at Harper University Hospital Director: Stephen D. Rioux, M.D. Co-Directors: Dilawer H. Abbas, M.D. Richard A. Lewis, M.D. Sindhu Ramchandren, M.D. Michael Shy, M.D.

25 East Lansing Springfield New Jersey Michigan State University Clinical St. John‘s Regional Health Center Newark Center Director: UMDNJ — University Hospital Director: Michael Luzecky, M.D. Co-Directors: Deborah F. Gelinas, M.D. St. Louis Jennifer Michaels, M.D. Farmington Hills Washington University School of John Bach, M.D. Michigan Institute for Neurological Medicine Stratford Disorders Director: Kennedy Memorial Hospital Co-Directors: Alan Pestronk, M.D.* Director: Martin I. Belkin, D.O. Montana Donald A. Barone, D.O. M. Eileen McCormick, D.O., F.A.A.P. Louis Rentz, D.O. Billings Tinton Falls David A. Simpson, D.O.* Billings Clinic The Rehabilitation Hospital of Tinton Co-Directors: Falls at HealthSouth Flint Mary Gaddy, M.D. Co-Directors: McLaren Regional Medical Center Lynn Fox Shenk, D.O. Neil R. Holland, M.D. Co-Directors: Judith F. Topilow, M.D. David A. Green, D.O. Great Falls Ronald Hunt, M.D. Benefis Hospital New Mexico Co-Directors: Albuquerque Grand Rapids William E. Henning, D.O. University of New Mexico Hospital Mary Free Bed Rehabilitation Hospital Bill J. Tacke, M.D. Director: Co-Directors: Sarah Youssof, M.D.* John F. Butzer, M.D. Missoula Deborah F. Gelinas, M.D.* Pediatric Specialty Clinic University of New Mexico Hospital Co-Directors: Director: Minnesota William E. Henning, D.O. Leslie Morrison, M.D. Duluth Bill S. Rosen, M.D. The Duluth Clinic New York Nebraska Co-Directors: Albany Wolcott S. Holt, M.D. Omaha Albany Medical Center Hospital Richard J. Kanoff, D.O. University of Nebraska Medical Center Director: Co-Directors: Matthew J. Murnane, M.D. Minneapolis J. Americo Fernandes, M.D. Fairview University Medical Center Gary Pattee, M.D. The Lewis Golub MDA/ALS Clinic at Director: Wayne Stuberg, Ph.D., P.T. St. Peter‘s Hospital John West Day, M.D., Ph.D. Director: Nevada Roberta Miller, M.D. St. Paul Las Vegas Gillette Children‘s Specialty Healthcare Amherst University of Nevada School of Director: Dent Neurologic Institute Medicine John West Day, M.D., Ph.D. Director: Co-Directors: Tomas Holmlund, M.D. Mississippi Donald W. Johns, M.D. Jackson David L. Ginsburg, M.D. Bronx Montefiore Medical Center University of Mississippi Medical Reno Co-Directors: Center Renown Institute for Neuroscience Emma C. Laureta, M.D. Director: Director: Alfred J. Spiro, M.D. V. V. Vedanarayanan, M.D., F.R.C.P.C. Timothy J. Louie, M.D. Brooklyn Missouri New Hampshire SUNY Downstate Medical Center Columbia Lebanon Co-Directors: University of Missouri Dartmouth-Hitchcock Medical Center Yaacov Anziska, M.D. Director: Co-Directors: Paul A. Pipia, M.D. Upinder K. Dhand, M.D. Jeffrey A. Cohen, M.D. Buffalo Joplin Brian D. Kossak, M.D. Erie County Medical Center St. John‘s Regional Medical Center Richard P. Morse, M.D. Director: Director: Linda Specht, M.D. Nicholas Silvestri, M.D. Taylor Bear, M.D.

26 New York Columbia University Medical Center Co-Directors: Darryl DeVivo, M.D.* Michio Hirano, M.D. Hiroshi Mitsumoto, M.D.* Joan and Sanford I. Weill Medical College of Cornell University Director: Dale J. Lange, M.D.* NYU Elaine A. and Kenneth G. Langone Medical Center Co-Directors: Mary Lynn Chu, M.D. Jeffrey M. Cohen, M.D. Winston-Salem Youngstown Port Jefferson Wake Forest University Baptist Medical Dean Martin Neuromuscular Clinic St. Charles Hospital and Rehabilitation Center Director: Center Director: Donald J. Tamulonis Jr., M.D. Co-Directors: James B. Caress, M.D. Scott McWilliams, M.D. Oklahoma Jennifer J. Semel, M.D. North Dakota Oklahoma City Fargo MDA Neuromuscular Center, Integris Rochester MeritCare Hospital Southwest Medical Center University of Rochester Medical Center Co-Directors: Director: Co-Directors: Richard Craig Bailly, M.D. Brent Beson, M.D. * Emma Ciafaloni, M.D. Susan L. Scarberry, M.D. Rabi Tawil, M.D. Oregon Charles A. Thornton, M.D.* Ohio Eugene Syracuse Cincinnati Sacred Heart Hospital SUNY Upstate Medical University Children’s Hospital Medical Center Director: Co-Directors: Director: Steven Goins, M.D. Brenda Wong, M.D., M.R.C.P., A.B.P.N. Carl J. Crosley, M.D. Medford Jeremy M. Shefner, M.D., Ph.D. * University of Cincinnati Medical Center Providence Medford Medical Center White Plains Director: Co-Directors: White Plains Hospital and Medical John Quinlan, M.D. Zakir Ali, M.D. Cornelia Byers, M.D. Center Cleveland Director: MetroHealth Medical Center Portland Stanley B. Holstein, M.D. Co-Directors: Oregon Health & Science University North Carolina Irwin B. Jacobs, M.D. Co-Directors: Marc David Winkelman, M.D. Edward Cupler, M.D. Chapel Hill Jau-Shin Lou, M.D., Ph.D.* University of North Carolina Hospitals Columbus Co-Directors: Ohio State University Hospital Portland James F. Howard Jr., M.D. Director: Shriners Hospital for Children Spencer G. Weig, M.D. John T. Kissel, M.D.* Co-Directors: Barry S. Russman, M.D. Charlotte Nationwide Children’s Hospital Michael D. Sussman, M.D. Carolinas Medical Center Director: Director: Jerry Mendell, M.D. Pennsylvania Benjamin R. Brooks, M.D.* Toledo Allentown Durham Toledo Children’s Hospital — Center Good Shepherd Rehabilitation Duke University Medical Center for Health Services Hospital Director: Director: Director: E. Wayne Massey, M.D. James A. Auberle, M.D. Terry D. Heiman-Patterson, M.D.

27 Wilkes-Barre Sioux Falls Geisinger Wyoming Valley Medical Neurology Associates Center Co-Directors: Director: Lisa C. Viola, D.O. James F. Hora, M.D., Ph.D. Todd A. Zimprich, M.D. Puerto Rico Tennessee Hato Rey Knoxville Hospital Auxilio Mutuo University of Tennessee Medical Director: Center Edward Ramos, M.D. Co-Directors: Randall G. Trudell, M.D. Ponce Brian F. Wiseman, M.D. Ponce School of Medicine Director: Memphis Carlos Otero, M.D. Wesley Neurology Clinic Director: San German Tulio Bertorini, M.D.* Danville Hospital de la Concepción Geisinger Medical Center Director: Nashville Director: Marla E. Jordan, M.D. Vanderbilt University Medical Center Scott M. Friedenberg, M.D. Director: San Juan Peter D. Donofrio, M.D.* Erie University of Puerto Rico Medical Northshore Neurosciences Sciences Texas Director: Director: Arlington Jeffrey J. Esper, D.O. Jose R. Carlo, M.D. Neurology Associates of Arlington Hershey Rhode Island Director: Hershey Medical Center Robert E. McMichael, M.D. Providence Co-Directors: Rhode Island Hospital Austin Anthony F. Giordano, M.D. Co-Directors: Specially for Children Zachary Simmons, M.D. James Gilchrist, M.D. Co-Directors: Philadelphia Karen Kerman, M.D. Jeffrey Kane, M.D. The Children’s Hospital of Philadelphia Michael Reardon, M.D. South Carolina Co-Directors: Corpus Christi Carsten G. Bönnemann, M.D. Charleston Driscoll Children’s Hospital Richard S. Finkel, M.D. Medical University of South Carolina Co-Directors: Gihan I. Tennekoon, M.D. Co-Directors: Mary Noreen Herring, M.D. Gary Bobele, M.D Drexel University College of Medicine Jerome E. Kurent, M.D. Carol C. Deline, M.D. Co-Directors: David Stickler, M.D. Dallas Anahita Deboo, M.D. Children’s Medical Center of Dallas Terry D. Heiman-Patterson, M.D.* Greenville Greenville Hospital System University Director: Hospital of the University of Medical Group Susan Iannaccone, M.D. Pennsylvania Co-Directors: Texas Neurology, P.A. Director: Addie S. Hunnicutt, M.D. Director: Shawn J. Bird, M.D. Augusto Morales, M.D. Daragh Heitzman, M.D. Pittsburgh Spartanburg University of Texas/SW Medical Center Children‘s Hospital of Pittsburgh Spartanburg Neurological Services Co-Directors: Co-Directors: Co-Directors: Jeffrey L. Elliott, M.D.* Hoda Z. Abdel-Hamid, M.D. Bogdan P. Gheorghiu, M.D. Sharon Nations, M.D. Mary Louise Russell, M.D. Robert A. Ringel, M.D. Gil I. Wolfe, M.D. University of Pittsburgh Medical South Dakota Fort Worth Center Rapid City Cook Children’s Medical Center Director: Regional Rehabilitation Institute Director: David Lacomis, M.D.* Director: Warren A. Marks, M.D. Matthew E. Simmons, M.D.

28 Harlingen Virginia West Virginia Valley Baptist Medical Center Charlottesville Morgantown Director: Kluge Children‘s Rehabilitation Center West Virginia University TBD Director: Co-Directors: Houston Christine M. Houlihan, M.D. Laurie Gutmann, M.D. Margaret E. Jaynes, M.D. The MDA Neuromuscular Clinic University of Virginia Hospital Co-Directors: Co-Directors: Wisconsin Stanley H. Appel, M.D.* Ted M. Burns, M.D. Green Bay Ericka P. Simpson, M.D. Lawrence H. Phillips II, M.D. St. Vincent Hospital/Prevea Clinic Texas Children’s Hospital Norfolk Co-Directors: Director: Sentara Norfolk General Hospital Terence S. Edgar, M.D. Timothy Lotze, M.D. Director: Prasad Kanneganti, M.D. Lubbock L. Matthew Frank, M.D. John Taylor, D.O. Covenant Health System Richmond Madison Co-Directors: Children‘s Hospital Dean Neurological Institute and Spine Bachar Al-Alami, M.D., P.A. Co-Directors: Center Ahmad Sabouni, M.D. Charles M. Dillard, M.D. Co-Directors: Roger Wolcott, M.D. Eugenio A. Monasterio, M.D. Oliver Ni, M.D. Round Rock Jean E. Teasley, M.D. Michael Snyder, M.D. Round Rock Medical Center Richmond University of Wisconsin Hospital & Director: Medical College of Virginia Clinics Adam D. Horvit, M.D. Co-Directors: Co-Directors: San Antonio Hamid Sadeghian, M.D. Barend P. Lotz, M.D.* University of Texas Health Science Scott A. Vota, D.O. Andrew J. Waclawik, M.D. Center Washington Marshfield Director: Marshfield Clinic Olympia Carlayne Jackson, M.D.* Director: Providence/St. Peter Hospital Rodney Sorensen, D.O. Sherman Director: Texoma Neurology Associates Greg Carter, M.D. Milwaukee Director: Children‘s Hospital of Wisconsin Pasco Easwar M. Sundaram Jr., M.D. Director: Physicians Health Center Safwan Jaradeh, M.D. Waco Director: Hillcrest Family Health Clinic Donald G. Dicken, M.D., P.C. Froedtert Memorial Lutheran Hospital Director: Co-Directors: Seattle George Mark Schwartze, M.D. Alexandru C. Barboi, M.D. Seattle Children‘s Hospital Wendy L. Peltier, M.D. Wichita Falls Director: The Neurology Center Susan D. Apkon, M.D. Director: University of Washington Medical Stephen Farmer, D.O. Center Utah Co-Directors: Salt Lake City Thomas Bird, M.D. University of Utah Medical Center George H. Kraft, M.D. Director: Michael Weiss, M.D.* Mark B. Bromberg, M.D., Ph.D.* Spokane Vermont Shriners Hospital for Children Burlington Co-Directors: Fletcher Allen Health Care Center Sharon Genung, M.D. Director: James Reggin, M.D. Rup Tandan, M.D. St. Luke‘s Rehabilitation Institute Director: Vivian M. Moise, M.D.

29 MDA/ALS Centers (As of July 2011) People with ALS may be seen at any of MDA’s 200 clinics.

Phoenix, Ariz. Baltimore, Md. Portland, Ore. MDA/ALS Center MDA/ALS Center MDA/ALS Center St. Joseph’s Hospital and Medical Johns Hopkins University School of Oregon Health & Science University Center Medicine Philadelphia, Pa. Tucson, Ariz. Boston, Mass. MDA/ALS Center of Hope MDA/ALS Center MDA/ALS Center Drexel University College of Medicine University Physicians Healthcare Massachusetts General Hospital Pittsburgh, Pa. Hospital Worcester, Mass. MDA/ALS Center Little Rock, Ark. MDA/ALS Center University of Pittsburgh Medical Center MDA/ALS Center University of Massachusetts Medical Memphis, Tenn. University of Arkansas for Medical Center MDA/ALS Center Sciences Farmington Hills, Mich. Wesley Neurology Los Angeles, Calif. MDA/ALS Center Nashville, Tenn. Jerry Lewis MDA/ALS Clinical and Michigan Institute for Neurological MDA/ALS Center Research Center Disorders Vanderbilt University Medical Center University of Southern California Grand Rapids, Mich. Dallas, Texas MDA/ALS Center MDA/ALS Center MDA/ALS Center University of California, Los Angeles Mary Free Bed Rehabilitation Hospital University of Texas/Southwestern Orange, Calif. St. Louis, Mo. Medical Center MDA/ALS Center MDA/ALS Center Houston, Texas University of California, Irvine Washington University School of MDA/ALS Center Medicine San Francisco, Calif. Methodist Neurological Institute MDA/ALS Center Albuquerque, N.M. San Antonio, Texas University of California San Francisco MDA/ALS Center MDA/ALS Center Medical Center University of New Mexico Health University of Texas Health Science Sciences Center Forbes Norris MDA/ALS Research Center Center New York, N.Y. Salt Lake City, Utah California Pacific Medical Center The Eleanor and Lou Gehrig MDA/ALS MDA/ALS Center Research Center Aurora, Colo. University of Utah MDA/ALS Center MDA/ALS Center Seattle, Wash. University of Colorado Joan and Sanford I. Weill Medical MDA/ALS Center College of Cornell New Britain, Conn. University of Washington Medical MDA/ALS Center Rochester, N.Y. Center Hospital for Special Care MDA/ALS Center Madison, Wis. University of Rochester Medical Center New Haven, Conn. MDA/ALS Clinical Research Center MDA/ALS Center Syracuse, N.Y. University of Wisconsin Hospital & Yale University MDA/ALS Center Clinics SUNY Upstate Medical University Miami, Fla. Kessenich Family MDA/ALS Center Charlotte, N.C. University of Miami MDA/ALS Center Carolinas Medical Center Atlanta, Ga. MDA/ALS Center Columbus, Ohio Emory University School of Medicine MDA/ALS Center Ohio State University Chicago, Ill. MDA/ALS Center Oklahoma City, Okla. University of Illinois at Chicago MDA/ALS Center Integris Southwest Medical Center Kansas City, Kan. MDA/ALS Center University of Kansas Medical Center 30 Officers (Officers serve one-year terms beginning July 15, 2011) Chairman of the Board Vice Chairman of the Executive Assistant Secretaries R. Rodney Howell, M.D. Committee Christina C. Kennedy Miami, Fla. Lois R. West Gail Schmertz Kerner, Esq. Hilton Head Island, S.C. Tucson, Ariz. President & CEO Gerald C. Weinberg Secretary Assistant Treasurers Tucson, Ariz. Timmi Masters Stephen Evans, C.P.A. Beverly Hills, Calif. Jodi Walters Chairman of the Executive Tucson, Ariz. Committee Treasurer Olin F. Morris Suzanne Lowden Memphis, Tenn. Las Vegas, Nev.

Board of Directors (Board members serve one-year terms beginning July 15, 2011)

Stanley H, Appel, M.D. Daniel G. Fries Timmi Masters * Gerald C. Weinberg Houston, Texas New York, N.Y. Beverly Hills, Calif. Tucson, Ariz. * Robert M. Bennett * R. Rodney Howell, M.D. Maureen McGovern * Lois R. West Los Angeles, Calif. Miami, Fla. Beverly Hills, Calif. Hilton Head Island, S.C. Bart Conner The Honorable Brad Henry * Olin F. Morris Norman, Okla. Edmond, Okla. Memphis, Tenn. Directors Emeriti: Benjamin F. Cumbo III Dave Hutton Edward M. Nigro Leon I. Charash, M.D. Upper Marlboro, Md. Valencia, CA Las Vegas, Nev. Hicksville, N.Y. Harold C. Crump Louis M. Kunkel, Ph.D. Christopher J. Rosa, Ph.D. Joseph S. DiMartino St. Paul, Minn. Boston, Mass. New York, N.Y. New York, N.Y. Steve Farella * Suzanne Lowden Charles Schoor, Esq. New York, N.Y. Las Vegas, Nev. Los Angeles, Calif. (* Executive Committee Members)

Translational Research Advisory Committee (Members as of July 2011)

Stanley H. Appel, M.D. John E. Howell Jeffery D. Rothstein, M.D., Ph.D. Methodist Neurological Institute Prometheus Partners Holdings Johns Hopkins University Houston, Texas Fairfax Station, Va. Baltimore, Md. Cristina Csimma, PharmD, M.H.P. Louis M. Kunkel, Ph.D. Charles Thornton, M.D. Independent consultant Harvard University/Children’s Hospital University of Rochester Lincoln, Mass. Boston, Mass. Rochester, N.Y. Merit Cudkowicz, M.D. Elizabeth McNally, M.D., Ph.D. Massachusetts General Hospital University of Chicago Boston, Mass. Chicago, Ill. Kenneth H. Fischbeck, M.D. John D. Porter, Ph.D. National Institutes of Neurological National Institutes of Neurological Disorders and Stroke Disorders and Stroke Bethesda, Md. Bethesda, Md.

31 Medical Advisory Committee (Members as of July 2011)

* Stanley H. Appel, M.D. Michio Hirano, M.D. Sally Nelson, Ph.D. Methodist Hospital Columbia University University of Colorado Houston, Texas New York, N.Y. Denver, Colo. Robert Baloh, M.D. James F. Howard, M.D. M. Kerry O’Banion, M.D., Ph.D. Washington University University of North Carolina University of Rochester St. Louis, Mo. Chapel Hill, N.C. Rochester, N.Y. Mark Bromberg, M.D., Ph.D. Joe Kornegay, D.V.M., Ph.D. Michael Shy, M.D. University of Utah University of North Carolina Wayne State University Salt Lake City, Utah Chapel Hill, N.C. Detroit, Mich. Thomas Crawford, M.D. Se-Jin Lee, M.D., Ph.D. Charles Thornton, M.D. Johns Hopkins University Johns Hopkins University University of Rochester Baltimore, Md. Baltimore, Md. Rochester, N.Y. Merit Cudkowicz, M.D., M.Sc. Katherine Mathews, M.D. Ex officio member: Massachusetts General Hospital University of Iowa R. Rodney Howell, M.D. Boston, Mass. Iowa City, Iowa University of Miami Miami, Fla. Heather Durham, Ph.D. Hiroshi Mitsumoto, M.D. Montreal Neurological Institute Columbia Presbyterian Medical Center Montreal, Quebec, Canada New York, N.Y. Richard Finkel, M.D. Kanneboyina Nagaraju, D.V.M, Ph.D Children’s Hospital of Philadelphia Children’s National Medical Center Philadelphia, Pa. Washington, D.C. (* Chairman)

Scientific Advisory Committee (Members as of July 2011)

* Louis M. Kunkel, Ph.D. Michael King, Ph.D. Bradley Olwin, Ph.D. Children’s Hospital — Boston Thomas Jefferson University University of Colorado Boston, Mass. Philadelophia, Pa. Boulder, Colo. John Adelman, Ph.D. Rashmi Kothary, Ph.D. Robin Parks, Ph.D. Oregon Health Sciences University Ottawa Health Research Institute Ottawa Health Research Institute Portland, Ore. Ottawa, Ontario, Canada Ottawa, Ontario, Canada Kurt Beam, Ph.D. Christian Lorson, Ph.D. Grace Pavlath, Ph.D. University of Colorado University of Missouri Emory University Denver, Colo. Columbia, Mo. Atlanta, Ga. Stephen Cannon, M.D. Gordon Lutz, Ph.D. John Ravits, M.D. University of Texas Southwestern Drexel University Virginia Mason Medical Center Medical Center Philadelphia, Pa. Seattle, Wash. Dallas, Texas Giovanni Manfredi, M.D., Ph.D. Melissa Spencer, Ph.D. Jeffrey Chamberlain, Ph.D. Weill Cornell Medical College University of California, Los Angeles University of Washington New York, N.Y. Los Angeles, Calif. Seattle, Wash. Elizabeth McNally, M.D., Ph.D. Maurice Swanson, Ph.D. Emanuela Gussoni, Ph.D. University of Chicago University of Florida Children’s Hospital Boston Chicago, Ill. Gainesville, Fla. Boston, Mass. Jeffrey Milbrandt, M.D., Ph.D. Ex officio member: Kenneth Hensley, Ph.D. Washington University R. Rodney Howell, M.D. University of Toledo St. Louis, Mo. University of Miami Toledo, Ohio Miami, Fla. Carlos Moraes, Ph.D. Bernard Jasmin, Ph.D. University of Miami University of Ottawa Miami, Fla. Ottawa, Ontario, Canada

(* Chairman) 32 National Vice Presidents (VPs serve one-year terms beginning July 15, 2011) James F. Ackerman Gary R. Chartrand Fred Greiner Bruce A. Lucia Indianapolis, Ind. Jacksonville, Fla. Boone, Iowa Atlanta, Ga. Max Adler John Clark John E. Haire Nigel Lythgoe Beverly Hills, Cailf. Clinton, N.C. New York, N.Y. Hollywood, Calif. Eric Affeldt Ginny Clements Derrick Hall Scott Masterson Dallas, Texas Tucson, Ariz. Phoenix, Ariz. New York, N.Y. William S. Anderson Nadia Comaneci James Halpin Hiroshi Mitsumoto, M.D. Albuquerque, N.M. Norman, Okla. New York, N.Y. New York, N.Y. Robin Arnold Norm Crosby Rusty Hardin Dana Moeller Purchase, N.Y. Los Angeles, Calif. Houston, Texas Phoenix, Ariz. Ty Ballou John F. Crowley Timothy A. Hawk Mark Moeller Pittsburgh, Pa. Cranbury, N.J. Conroe, Texas St. Paul, Minn. Brandon Barash Edward L. Davis, Ed.D. Kenneth W. Heath Jeffrey S. Moorad Los Angeles, Calif. Reston, Va. New York, N.Y. Newport Beach, Calif. Michael A. Barras Linda Davis Ron Hemelgarn Natalie Morales Lafayette, La. Hermitage, Tenn. Toledo, Ohio New York, N.Y. Lynn Beall Peter Dectis Tom Henry Ted Moudis, A.I.A. St. Louis, Mo. Allentown, Pa. St. Louis, Mo. New York, N.Y. Candace K. Beinecke Wayne Denningham Daniel F. Hilferty S. Paul Musco New York, N.Y. Fort Worth, Texas Broomhall, Pa. Santa Ana, Calif. Mike Bellotti Frank DiBella, C.P.A. Jacob “Jake” Jabs Robert J. Myers Eugene, Ore. Whittier, Calif. Englewood, Colo. Ankeny, Iowa Gordie Boucher Thomas R. Donahue Daryl Johnston Michael P. Neary Greenfield, Wis. Washington, D.C. Dallas, Texas New York, N.Y. Tom Bovis Gary R. Drewing Wynonna Judd Augie Nieto Providence, R.I. Columbia, Mo. Nashville, Tenn. Corona del Mar, Calif. Don Breckenridge Jr. Thomas Ehlmann Tariq Khan Nancy O’Dell Webster Groves, Mo. Fort Worth, Texas Rockville Centre, N.Y. Pasadena, Calif. Bill Breetz William Emmons Randy Kibler Tom Odisho Shenandoah, Texas Fort Worth, Texas Charlotte, N.C. Charenton, La. DeLight Breidegam Jr. W. King Engel, M.D. Bill Klesse Tony Orlando Lyon Station, Pa. Los Angeles, Calif. San Antonio, Texas Branson, Mo. Todd Bresnahan Charles Fazzino Mario Kreutzberger James P. Prather Phoenix, Ariz. New Rochelle, N.Y. Miami, Fla. Las Vegas, Nev. Julie Brinks Jay Feeley John A. Krol John Quinlan, M.D. Tucson, Ariz. Plantation, Fla. Wilmington, Del. Cincinnati, Ohio James M. Brown Kara Fox-LaRose Evan Lamberg Charo Rasten Oklahoma City, Okla. Wilkes-Barre, Pa. New York, N.Y. Los Angeles, Calif. Anthony Cammarata Jr. Stephen J. Furnary Juan Antonio Larrea Larree Renda Hudson, N.J. New York, N.Y. San Juan, Puerto Rico Pleasanton, Calif. Tony Cammerata Joe Gentile Jr. Eric Lassberg Jerry Reynolds Berea, Ohio Dunmore, Pa. Austin, Texas Carrollton, Texas Alex Cappello Neil M. Golub Patricia Laus Thomas Rice Santa Monica, Calif. Schenectady, N.Y. Manasquan, N.J. New York, N.Y. Jann T. Carl Richard Graziano Paul W. Lowden Petra Robinson Pasadena, Calif. Hartford, Conn. Las Vegas, Nev. San Marcos, Calif.

33 National Vice Presidents

Tom Robinson Mike Sheehan Alison Sweeney Salisbury, N.C. Chicago, Ill. Hollywood Hills, Calif. Fredric V. Rolando John Shoop Tom Thomas Washington, D.C. Milton, Pa. Rancho Dominguez, Calif. Juan José Román Lynn Shostack Raymond M. Tierney III San Juan, Puerto Rico New York, N.Y. New York, N.Y. Lon Rosenberg Robert Sigel Justin Tuck Landover, Md. Westborough, Mass. Hackensack, N.J. Lewis P. Rowland, M.D. Alan Simonini Lisa Marie Utasi New York, N.Y. Charlotte, N.C. New York, N.Y. Mike Rowlett Chris Snee Kevin Urban Irving, Texas Franklin Lakes, N.J. Jonesville, N.C. Harold A. Schaitberger Aniceto Solares Phil Villapiano Washington, D.C. San Juan, Puerto Rico Rumson, N.J. John Seabers Bill Spence Bryan Ward San Antonio, Texas Crystal Lake, Ill. Pittsburgh, Pa. Rick Seithel Jennifer Smith Stepanek, Ed Williams Overland Park, Kan. Ph.D. Fairless Hills, Pa. Rockville, Md. Bert Selva Ace Young Walnut, Calif. Carol Stillwell Sherman Oaks, Calif. Edison, N.J.

Task Force on Public Awareness (Members serve one-year terms beginning July 15, 2011)

William W. Altaffer, Esq. Rob Roozeboom Tucson, Ariz. Sheldon, Iowa Aaron Bates, Esq. Christopher Rosa, Ph.D. Orlando, Fla. Flushing, N.Y. Jan Blaustone David A. Sheffield, Esq. Nashville, Tenn. Silsbee, Texas Adnan Gill Bradley R. Stephenson, Esq. Rancho Palos Verdes, Calif. San Antonio, Texas Nicholas A. Johnson L. Vance Taylor Waltham, Mass. Oxon Hill, Md. Frank Lombardi Angela Wrigglesworth Leesburg, Va. Houston, Texas Michael Neufeldt New Berlin, Wis.

Christopher Rosa, Ph.D.

34 Report From the Treasurer

Powered by the commitment of our donors, dent auditors, issued an unqualified opinion the Muscular Dystrophy Association’s research on our financial statements. Following are and services programs provided steady condensed statements of financial position beacons of hope to individuals and families and activities that were derived from our 2010 affected by neuromuscular diseases in 2010. audited financial statements. For a copy of the complete financial statements and the The Association spent more than 77 cents of report of our independent auditors, contact every dollar on program services in 2010. Patient the Finance Department at MDA National and community services accounted for approxi- Headquarters, 3300 E. Sunrise Drive, Tucson, mately 43 cents; research, 21 cents; and profes- Arizona, 85718. sional and public health education, 13 cents.

MDA volunteers, sponsors, researchers and staff worked tirelessly to advance the fight against more than 40 forms of muscle disease. Confronted by a weak economy throughout 2010, the Association continued to Suzanne Lowden review and streamline costs, while implement- Treasurer ing effective, creative strategies to maintain Muscular Dystrophy Association and enhance the quality of its programs. July 16, 2011

The financial statements of MDA for the year ended December 31, 2010, were prepared in accordance with accounting principles generally accepted in the United States for not-for-profit organizations. Ernst & Young LLP, our indepen-

35 Statement of Financial Position December 31, 2010

Assets Cash and cash equivalents $ 21,809,108 Contributions receivable, net of allowance for 5,917,002 doubtful accounts of $450,000 Prepaid expenses and other assets 3,158,123 Investments 75,354,127 Fixed assets, net 14,984,610 Total assets $ 121,222,970

Liabilities and net assets Liabilities: Accounts payable and accrued expenses $ 7,779,948 Research awards, grants and fellowships payable 26,132,145 Pension and post-retirement plan obligations 20,039,234 Total liabilities 53,951,327

Net assets: Unrestricted: Available for program and supporting services 51,129,412 Net investment in fixed assets 14,984,610 Temporary restricted 1,157,621 Total net assets 67,271,643 Total liabilities and net assets $ 121,222,970

Financial Position 67,271,643 121,222,970

Total assets

Total net assets

Total liabilities

53,951,327

36

Revenue

6,191,359 815,571 23,331,991 246,742 8,437,577

139,052,405

Special events, net - 78.1% Indirect public support - 0.5%

Contributions - 13.1% Investment income and other revenue - 3.5%

Bequests and legacies - 4.7% Net assets released from restrictions - .1%

Expenses 23,222,445 14,407,973

26,106,112

77,263,235 38,743,491

Patient and community services, net - 43% Research - 21.6%

Professional and public health education - 12.9% Fundraising - 14.5%

Management and general - 8% Statement of Activities — Revenue Year Ended December 31, 2010

Public support: Received directly: Special events, including Telethon $ 173,367,689 Less fundraising direct benefit costs (34,315,284) Special events, net 139,052,405 Contributions Financial Position 23,331,991 Bequests and legacies 8,437,577 67,271,643 Total received directly 170,821,973 121,222,970

Received indirectly: Total assets Combined Federal Campaign and Combined Health Appeals 815,571 Total net assets Total revenue from the public 171,637,544 Investment income and other revenue 6,191,359 Total liabilities Total 53,951,327unrestricted revenue $ 177,828,903 Net assets release from restrictions 246,742 Total unrestricted revenue and support $ 178,075,645

Revenue

6,191,359 815,571 23,331,991 246,742 8,437,577

139,052,405

Special events, net - 78.1% Indirect public support - 0.5%

Contributions - 13.1% Investment income and other revenue - 3.5%

Bequests and legacies - 4.7% Net assets released from restrictions - .1%

37 Expenses 23,222,445 14,407,973

26,106,112

77,263,235 38,743,491

Patient and community services, net - 43% Research - 21.6%

Professional and public health education - 12.9% Fundraising - 14.5%

Management and general - 8% Financial Position 67,271,643 121,222,970

Total assets

Total net assets

Total liabilities

53,951,327 Statement of Activities — Expenses Year Ended December 31, 2010

Program services: Revenue Patient and community services, net of third-party reimbursements of $854,570 $ 77,263,235 6,191,359 815,571 23,331,991 246,742 Research 38,743,491 8,437,577 Professional and public health education 23,222,445 Total program services 139,229,171

Supporting services: Fundraising 26,106,112 Management and general 14,407,973 Total supporting services 40,514,085 Total expenses 179,743,256 139,052,405

Decrease in unrestricted net assets from operations (1,667,611) Special events, net - 78.1% Indirect public support - 0.5% Changes in unrecognized benefit plan costs (5,314,996) Contributions - 13.1% Investment income andDecrease other revenue in unrestricted - 3.5% net assets (6,982,607)

Bequests and legacies - 4.7% Net assets released fromNet restrictions assets released - .1% from restrictions (246,742) Decrease in net assets (7,229,349) Net assets, beginning of year 74,500,992 Net assets, end of year $ 67,271,643 Expenses 23,222,445 14,407,973

26,106,112

77,263,235 38,743,491

Patient and community services, net - 43% Research - 21.6%

Professional and public health education - 12.9% Fundraising - 14.5%

Management and general - 8%

38 Join Our Muscle Team®!

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