Band-aid for Muscular Dystrophy?

May 25, 2009 11:25:18 AM PDT
Muscular dystrophy (MD) is a disorder that gradually degenerates the body's muscles which control movement. It is an inherited disease caused by a genetic mutation, preventing the production of proteins needed for healthy muscles. MD progressively weakens voluntary muscles, and the different types of MD vary with age of onset and rate of progression. Symptoms include muscle weakness, lack of coordination, and progressive crippling or loss of mobility. DUCHENNE MUSCULAR DYSTROPHY: Duchenne muscular dystrophy (DMD) is the most severe form of the disease, mostly affecting young boys, and is the most common from of MD that affects children. DMD first affects the muscles in the pelvis, upper arms and upper legs. Symptoms often begin between the ages of four and six, including frequent falls, large calf muscles, weakness in lower leg muscles, and difficulty getting up from lying down.

Children with DMD are recommended to see a heart and lung specialist, as the heart may also be affected by the disorder. With progression of the disease, the lungs may also weaken, and a ventilator may be needed to assist with breathing. Most children with DMD are unable to walk by late childhood, often requiring a wheelchair by age 10 or 12. Patients with DMD often do not survive past their late teens or early adulthood.

TREATMENT: There is no treatment to cure any form of muscular dystrophy. The National Institute of Neurological Disorders and Stroke states helpful treatments include physical therapy, respiratory therapy, speech therapy, orthopedic appliances used for support, and corrective orthopedic surgery.

As MD weakens the muscles, fixations develop in the joints, tendons shorten, and flexibility decreases. Physical therapy can help delay the formation of fixations in the joints. Range of motion exercises are practiced during physical therapy, benefitting flexibility.

DRUG TREATMENTS: The only medication prescribed to patients with DMD is prednisone, which can improve muscle strength. One promising drug about to debut in human trials has been shown to cover up the gene mutation that causes DMD in dogs and encourage the production of the protein missing in patients with the disease. The drug is a "cocktail" of laboratory-developed compounds. If the drug is shown to be effective and safe in humans, it could benefit up to 90 percent of people with DMD.

FOR MORE INFORMATION, PLEASE CONTACT:

Genetic Counseling
Children's National Medical Center
(202) 476-4925
http://www.childrensnational.org


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