Last Updated: 6/08

MDA Fact Sheet

What Is MDA?

The Muscular Dystrophy Association is a voluntary national health agency — a dedicated partnership between scientists and concerned citizens aimed at conquering neuromuscular diseases that affect more than a million Americans. Your local MDA office, one of more than 200 nationwide, is available to serve anyone with one of the diseases in the Association’s program. Energized by its national chairman, Jerry Lewis, who has been the Association's number-one volunteer for more than 50 years, MDA today is one of the world's leading voluntary health agencies fostering research and medical care. Programs available through your local MDA office are funded almost entirely by individual private contributors. The Association receives no government grants.

How Did MDA Get Started?

As late as 1950, very little was being done to combat muscular dystrophy. In that year, a small group of parents whose youngsters had muscular dystrophy decided to do something about this, and MDA was born. Those parents firmly believed that there are no incurable diseases, only diseases for which no treatments have yet been found. That basic philosophy has animated MDA ever since.

What Diseases Is MDA Striving To Defeat?

Muscular Dystrophies: There is no single disease called muscular dystrophy. The term designates a group of hereditary muscle-destroying disorders, which vary in inheritance pattern, age of onset, initial muscles attacked and rate of progression.

Motor Neuron Diseases: This is a group of progressive diseases chiefly characterized by degeneration of the motor nerve cells in the spinal cord. They include amyotrophic lateral sclerosis (ALS) and several forms of spinal muscular atrophy (SMA).

Diseases of the Neuromuscular Junction: Myasthenia gravis is an autoimmune disease characterized by fluctuating weakness resulting from a failure in the transmission of signals from nerves to muscles. The disease initially affects eye movement, facial expression, chewing, swallowing and respiration, and can later affect arm and leg muscles. Lambert-Eaton (myasthenic) syndrome is associated with initial weakness of the muscles of the shoulders and thighs. Congenital myasthenic syndromes result from genetic defects.

Metabolic Disease of Muscle: These progressive diseases affect voluntary muscles and are characterized by inherited chemical deficiencies — usually of a specific enzyme such as phosphorylase (McArdle disease), acid maltase (Pompe disease) or carnitine palmityl transferase.

Diseases of Peripheral Nerves: These diseases affect the peripheral nervous system, causing progressive weakness and atrophy of muscles. Friedreich’s ataxia is characterized by shaky movements and unsteadiness due to degeneration of both peripheral nerves and certain nerve cells in the brain and spinal cord. Charcot-Marie-Tooth disease is characterized by progressive weakness of the feet, lower legs, hands and forearms, and a mild loss of sensation in the limbs. It results from degeneration of peripheral nerves that control muscle contraction and sensation.

The Inflammatory Myopathies: This group of potentially debilitating diseases is characterized by progressive weakness and inflammation of skeletal muscle and is attributed to disturbances in the immune system. Polymyositis can cause weakness of the pelvis, shoulders and limbs, as well as difficulty in swallowing. Dermatomyositis can cause similar symptoms and is accompanied by a rash and other changes in the skin. Inclusion-body myositis causes atrophy of the arms and legs, usually in people over 50.

Myopathies Due to Endocrine Abnormalities: Disorders of the thyroid gland can sometimes affect muscle function. Hyperthyroid myopathy is characterized by muscle weakness and muscle wasting. Hypothyroid myopathy causes delayed muscle relaxation, stiffness and painful cramps.

Other Myopathies: This is a group of unrelated genetic muscle disorders, usually named for peculiarities seen in biopsies of muscle tissue. Central core disease, usually apparent at birth, causes skeletal deformities and diffuse weakness. Nemaline myopathy, also usually apparent at birth, results in loss of muscle tone and progressive weakness in limb and trunk muscles. Myotubular myopathy causes poor muscle tone at birth and varying degrees of weakness in eye, facial, neck and limb muscles.

What Does MDA Do?

MDA works in your community to combat neuromuscular diseases through (1) basic and applied scientific investigation, (2) local comprehensive programs of medical and support services, and (3) widespread professional and public health education. Thanks to the Jerry Lewis MDA Labor Day Telethon (brought to you and thousands of other viewers in your area by a nearby “Love Network” TV station), and to many other local fundraising projects involving all sectors of the community, MDA has been able to organize and maintain a variety of programs and services.

Worldwide Research: Grants are awarded to physicians and scientists at hospitals, universities, nonprofit institutions and biotechnology companies in the United States and abroad who are seeking the causes of, and treatments and cures for, any of the disorders in MDA’s program.

In 2007, MDA allocated $37 million for research, funding some 350 projects. Careful review by MDA’s medical, scientific and translational research advisory committees ensures resources are allocated to achieve maximum results.

MDA-supported scientists have begun the first U.S. trial of gene therapy for Duchenne muscular dystrophy by injecting a miniaturized version of the gene for the muscle protein dystrophin into an arm muscle of boys with this disorder. This phase 1 safety study will pave the way for further gene therapy trials in this and other MDA-covered disorders.

MDA also is supporting development of compounds that allow muscle cells to skip defective parts of a gene or ignore molecular “stop signs.” Both these strategies allow cells to produce functional protein molecules even when a genetic flaw is present.

Tests of an enzyme replacement therapy for the metabolic muscle disorder acid maltase deficiency, derived from an MDA-funded project, had lifesaving results for affected infants, and resulted in FDA approval in 2006 of the first definitive treatment for a genetic disease in MDA’s program.

In ALS (Lou Gehrig’s disease), MDA is supporting a $36 million industrial-scale research-and-development program to identify potential ALS treatments through the ALS Therapy Development Institute in Cambridge, Mass. MDA also is funding the development of a compound to block the synthesis of a toxic protein in familial ALS. MDA ALS Centers are the sites of dozens of trials of new and existing medications and other treatments.

Nationwide Services: MDA maintains the most comprehensive services program of any voluntary health agency, helping individuals and their families meet the challenges imposed by chronic, progressive diseases. Included among these services is a network of outpatient clinics; assistance with the purchase and repair of wheelchairs, leg braces and communication devices; equipment loan closets; support groups; youth summer camp programs; flu shots; and resource referral. In 2007, MDA allocated $87 million for patient and community services.

Network of Clinics: MDA clinics offer an interdisciplinary team approach toward initial diagnosis and follow-up care. Individuals affected by any of the diseases in MDA’s program have access to a nationwide network of 220 hospital-affiliated clinics staffed by top health professionals. These experts can advise about all aspects of medical management of a disease, including occupational, speech, respiratory and physical therapies. While all MDA clinics serve people with amyotrophic lateral sclerosis (ALS), MDA has designated 36 facilities as MDA/ALS centers because of the amount of ALS research taking place there and the vast experience of the medical staff in dealing with this disease.

Summer Camps: MDA provides youngsters in your community with summer camping activities geared to their special needs. In 2007, MDA sponsored 92 camp sessions for more than 4,400 campers. MDA camps are staffed by health professionals who volunteer their services. Campers are assisted by volunteer counselors, who often continue their friendships with campers on a year-round basis.

Professional and Public Health Education: MDA seeks to provide the power of information to those living with muscle diseases, and to increase knowledge and awareness of these diseases among scientists, physicians, nurses, therapists and the general public. The Association publishes and distributes a wide array of educational materials, including the award-winning quarterly magazine Quest, and dozens of informational and scientific booklets, some in Spanish. These publications also are available on MDA’s comprehensive Web site at www.mda.org. (A Spanish-language Web site is located at www.mdaenespanol.org.) In addition, MDA regularly convenes international scientific meetings about disease research and holds conferences of MDA clinic directors and their associates.

MDA Accreditation

MDA is the first nonprofit organization honored with the American Medical Association’s Lifetime Achievement Award “for significant and lasting contributions to the health and welfare of humanity.”

In your state, county and city, MDA operates in accordance with all ordinances and regulations governing fundraising activities and has never been denied a license anywhere.

Who Supports MDA?

MDA’s sponsors include: ACOSTA Sales and Marketing Co.; Burger King; CITGO Petroleum Corp.; ClubCorp; Lowe’s Home Improvement; Convenience Store Industry; Dr Pepper/7 UP; ERA Real Estate; Harley-Davidson Motor Co.; International Association of Fire Fighters; International Health, Racquet & Sportsclub Association; Miller Brewing Co., National Association of Letter Carriers; Outback Steakhouse; RealNetworks; Safeway; 7-Eleven Franchisees; SuperValu/Albertsons; and Tall Cedars of Lebanon of North America.

National youth organizations supporting MDA include: DECA; Kappa Alpha Order; and Universal Cheerleaders and Dance Associations.

DISEASES IN MDA’S PROGRAM
The following diseases are included in MDA’s research and services programs: