MDA Telethon Funds Keep Research Progress, Clinics, Camps Going

Behind the fun and entertainment of the Muscular Dystrophy Association’s 2003 Jerry Lewis Labor Day Telethon Aug. 31-Sept. 1 is a serious purpose.

The 21½-hour Telethon, broadcast on some 200 “Love Network“ stations across the country, is MDA’s single largest fund-raising event. Last year’s Telethon yielded a record $58.3 million in donations and pledges to fund programs of research, medical and community services, and public health education.

This year, MDA’s allocating more than $32 million to its worldwide research program. Donations to the Telethon also help provide services for children and adults affected by more than 40 neuromuscular diseases, and fund professional and public health education and training.

“The exciting research progress, the tens of thousands of visits to MDA clinics, the thousands of kids who have the time of their lives at MDA summer camps — these are just some of the fruits of last year’s record-setting Telethon,” said Robert Ross, MDA president & CEO and executive producer of the Telethon. “By showing how much they care through their Telethon pledges and contributions, the American people have helped vitally to move our search for treatments and cures ahead, and to provide crucial services to people with neuromuscular diseases.”

About 75 cents of each dollar MDA spends goes directly to program services. A voluntary health agency, MDA relies almost entirely on private contributions and receives no government funds.

Research Progress

MDA-funded scientists have made scores of major advances that put treatments or cures for neuromuscular diseases in sight. In the past year, MDA researchers have:

·        Combined gene therapy with stem cell therapy to correct Duchenne muscular dystrophy in mice, and continued human trials of the antibiotic gentamicin, which may be effective in some children with DMD

·        Continued to support lifesaving human trials of enzyme replacement therapy for Pompe's disease, a metabolic disease of muscle that’s often fatal to infants

·        Assisted with development and testing of potential drug therapies for Lou Gehrig’s disease (amyotrophic lateral sclerosis), and located genetic flaws in a nerve cell supply line that could be a pivotal event leading to ALS

·        Set the stage for using gene therapy to treat spinal muscular atrophy (SMA)

·        Tested two methods of “gene silencing” in cells harboring the gene flaw that causes myotonic muscular dystrophy type 1, the most common form of MD in adults

·        Found that more than 70 percent of patients with myasthenia gravis responded positively to the transplant rejection prevention drug, CellCept

Comprehensive ServicesChildren and adults with neuromuscular diseases in communities nationwide benefit directly from public support of the Telethon. MDA is allocating more than $80 million this year to services for people with neuromuscular diseases, including:

·        Sending nearly 4,300 children to MDA summer camps, at no cost to their families

·        Registering some 70,000 visits to the 230 hospital-affiliated MDA clinics and 30 MDA/ALS centers across the country for diagnosis; follow-up treatment; physical, occupational and respiratory therapy; and counseling

·        Giving free flu vaccinations to help prevent potentially deadly respiratory complications

·        Offering financial assistance in the purchase of wheelchairs, leg braces and communication devices

·        Loaning out thousands of pieces of adaptive equipment such as hydraulic lifts, wheelchairs and hospital beds

·        Offering some 270 MDA-facilitated support groups and hosting nearly 100 online chat sessions each month

Professional And Public Education

Information and communication are powerful allies in the fight against neuromuscular diseases. Telethon donations help fund professional seminars and scientific conferences, as well as up-to-date informative booklets, care guides, videos, the award-winning Quest magazine, The MDA/ALS Newsletter and other presentations.

MDA’s Hispanic outreach program, led by MDA Vice President Mario Kreutzberger (alias Don Francisco), resulted in a dramatic increase in calls from Spanish speakers to MDA’s toll-free information hotline (800-572-1717). The Association’s Web sites (, and received millions of visits, provided thousands of answers to users’ questions, and hosted more than two dozen online clinical-research Q&A sessions featuring scientific and medical specialists.

— Adapted from the Muscular Dystrophy Association 2003 Digital Media Kit