Since 1995 - Non Profit Healthcare Advice

Muscular Dystrophy

04/06/2000 08:17AM

Question:

Can people with muscular dystrophy get any care or help from any kind of therapy? If so, which kind of therapy? If you could please enclose some additional information if possible. Thanks!

Answer:

Muscular dystrophy refers to a group of genetic diseases that cause progressive weakness and degeneration of the skeletal, or voluntary, muscles, which control movement. The muscles of the heart and some other involuntary muscles are also affected in some forms of muscular dystrophy, and a few forms involve other organs as well. There are many different types of muscular dystrophy that can affect both children and adults.

The treatment or management of muscular dystrophy is aimed mostly at treating the symptoms: exercise programs and physical therapy help to keep muscles moving; providing devices that help with walking (canes, powered wheelchairs), preventing scoliosis (curvature of the spine), and helping to maintain good lung capacity and breathing. There is no known cure at this time. Sometimes surgery is used to release muscles and prevent severe contractures (shortening) of muscles.

There are a number of experimental treatments that are being conducted in animals or in human trials that will hopefully find new treatments for these diseases.

Medications known as corticosteroids have been found to slow muscle destruction in some types of muscular dystrophy. However, these potent anti-inflammatory drugs, used for many conditions besides muscular dystrophy, can have serious side effects. Researchers are testing a new corticosteroid that may have fewer side effects. They are also studying how these drugs slow muscle destruction hoping to find a drug that would cause less side effects.

Initial experimental gene therapy trials in people – trying to replace dystrophin – the protein that is abnormal in muscular dystrophy – have been disappointing. Gene replacement with dystrophin minigenes is being investigated in animals, but not in humans yet.

There is an excellent website sponsored by the Muscular Dystrophy Association that will provide you with additional information.

For more information:

Muscular Dystrophy Association


Response by:

Case Western Reserve University Anne Matthews, R.N., Ph.D.
Director, Genetic Counseling and Family Studies

Center for Human Genetics



School of Medicine



Case Western Reserve University

Anne   Matthews, R.N., Ph.D.