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Muscular Dystrophy
The muscular dystrophies (MD) are a group of more than 30 genetic diseases characterized by progressive weakness and degeneration of the skeletal muscles that control movement. Some forms of MD are seen in infancy or childhood, while others may not appear until middle age or later. The disorders differ in terms of the distribution and extent of muscle weakness (some forms of MD also affect cardiac muscle), age of onset, rate of progression, and pattern of inheritance.Duchenne MD is the most common form of MD and primarily affects boys. It is caused by the absence of dystrophin, a protein involved in maintaining the integrity of muscle. Onset is between 3 and 5 years and the disorder progresses rapidly. Most boys are unable to walk by age 12, and later need a respirator to breathe. Girls in these families have a 50 percent chance of inheriting and passing the defective gene to their children. Boys with Becker MD (very similar to but less severe than Duchenne MD) have faulty or not enough dystrophin.Facioscapulohumeral MD usually begins in the teenage years. It causes progressive weakness in muscles of the face, arms, legs, and around the shoulders and chest. It progresses slowly and can vary in symptoms from mild to disabling.Myotonic MD is the disorder's most common adult form and is typified by prolonged muscle spasms, cataracts, cardiac abnormalities, and endocrine disturbances. Individuals with myotonic MD have long, thin faces, drooping eyelids, and a swan-like neck.
Treatment
There is no specific treatment to stop or reverse any form of MD. Treatment may include physical therapy, respiratory therapy, speech therapy, orthopedic appliances used for support, and corrective orthopedic surgery. Drug therapy includes corticosteroids to slow muscle degeneration, anticonvulsants to control seizures and some muscle activity, immunosuppressants to delay some damage to dying muscle cells, and antibiotics to fight respiratory infections. Some individuals may benefit from occupational therapy and assistive technology. Some patients may need assisted ventilation to treat respiratory muscle weakness and a pacemaker for cardiac abnormalities.
Prognosis
The prognosis for people with MD varies according to the type and progression of the disorder. Some cases may be mild and progress very slowly over a normal lifespan, while others produce severe muscle weakness, functional disability, and loss of the ability to walk. Some children with MD die in infancy while others live into adulthood with only moderate disability.
Research
The NINDS supports a broad program of research studies on MD. The goals of these studies are to understand MD and to develop techniques to diagnose, treat, prevent, and ultimately cure the disorder. The NINDS is a member of the Muscular Dystrophy Coordinating Committee (MDCC). For additional information, please visit:http://www.ninds.nih.gov/find_people/groups/mdcc/index.htm
View a list of studies currently seeking patients.
View more studies on this condition.
Read additional information from Medline Plus.
Organizations
Facioscapulohumeral Muscular Dystrophy (FSH) Society
Facilitates support groups, publishes a newsletter, organizes conferences and meetings, and awards research grants towards the prevention, cause and treatment of FacioScapuloHumeral muscular dystrophy worldwide. Provides public awareness of FSHD by providing information, referrals, education, and advocacy programs and services. Promotes collaborative research and collects and disseminates research information.
64 Grove Street
Watertown, MA 02472
Tel: 617-658-7877
Fax: 617-658-7879
Muscular Dystrophy Association
Voluntary health agency that fosters neuromuscular disease research and provides patient care funded almost entirely by individual private contributors. MDA addresses the muscular dystrophies, spinal muscular atrophy, ALS, Charcot-Marie-Tooth disease, myasthenia gravis, Friedreich's ataxia, metabolic diseases of muscle, and inflammatory diseases of muscle, for a total of more than 40 neuromuscular diseases.
3300 East Sunrise Drive
Tucson, AZ 85718-3208
Tel: 520-529-2000
800-572-1717
Fax: 520-529-5300
Muscular Dystrophy Family Fund
Provides services, resources, adaptive equipment, and home medical equipment to individuals with muscular dystrophy and their families to improve independence and quality of life. Focuses on meeting the day-to-day needs of individuals and families.
1033 Third Avenue SW
Suite 108
Suite 108
Carmel, IN 46032
Tel: 317-249-8488
Fax: 317-615-9140
Parent Project Muscular Dystrophy (PPMD)
Dedicated to ending Duchenne -- to accelerate research, advocate, demand optimal care for all young men, and educate the global community.
401 Hackensack Avenue, 9th Floor
Hackensack, NJ 07601
Tel: 800-714-KIDS (5437)
Fax: 201-944-9987
International Myotonic Dystrophy Organization
International foundation dedicated to improved management and treatment of Myotonic Dystrophy. Supports patients with information and services worldwide.
P.O. Box 1121
Sunland, CA 91041-1121
Tel: 818-951-2311
866-679-7954
National Institute of Child Health and Human Development (NICHD)
National Institutes of Health, DHHS
31 Center Drive, Rm. 2A32 MSC 2425
31 Center Drive, Rm. 2A32 MSC 2425
Bethesda, MD 20892-2425
Tel: 301-496-5133
Fax: 301-496-7101
Centers for Disease Control and Prevention (CDC)
U.S. Department of Health and Human Services
1600 Clifton Road, N.E.
1600 Clifton Road, N.E.
Atlanta, GA 30333
Tel: 800-311-3435
404-639-3311/404-639-3543
Cure CMD
Cure CMD’s mission is to bring research, treatments and in the future, a cure for Congenital Muscular Dystrophies. Cure CMD will achieve this mission by working globally together with dedicated parent, government and research advocates. By focusing on this mission, Cure CMD will find and fund high potential research and clinical trials. Success will be determined by clinical applications that improve the lives of those afflicted with CMD’s.
P.O. Box 701
Olathe, KS 66051
Tel: 1-866-400-3626
Coalition to Cure Calpain 3 (C3)
A voluntary health organization that provides support for promising research into finding treatments or a cure for limb-girdle muscular dystrophy, type 2A/calpainopathy (LGMD2A).
15 Compo Parkway
Westport, CT 06880
Tel: 203-221-1611
Fax: 734-668-4755
Jain Foundation
The Jain Foundation seeks to expedite development of a cure or therapy for Limb-girdle muscular dystrophy type 2B (LGMD2B) and Miyoshi Myopathy. In addition to educating physicians and patients about LBMD2B/Myoshi, the Foundation maintains a patient registry, offers help with diagnosis (e.g., funding dysferlin gene mutational analysis), funds and monitors research and progress, provides financial support to accelerate clinical trials, and encourages scientific collaboration.
2310 130th Ave NE
Suite B101
Suite B101
Bellevue, WA 98005
Tel: 425-882-1440
Fax: 425-658-1703
National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS)
National Institutes of Health, DHHS
31 Center Dr., Rm. 4C02 MSC 2350
31 Center Dr., Rm. 4C02 MSC 2350
Bethesda, MD 20892-2350
Tel: 301-496-8190
877-22-NIAMS (226-4267)