Muscular Dystrophy - Treatment






There are currently no cures for any form of muscular dystrophy. A few drugs have been found that slow the progress of some forms of MD. For example, prednisone (pronounced PRED-nih-zone), a corticosteroid (pronounced kor-tih-ko-STIHR-oid), slows the progress of DMD. Generally speaking, however, drugs have a limited and uncertain value in the treatment of MD.

The primary goal of treatment programs for MD is to prevent complications. The major complications are decreased ability to move on one's own, contractures, scoliosis, heart defects, and respiratory problems.

Physical Therapy

Regular stretching exercises help prevent or delay contractures. Braces may be used to help support ankles, feet, legs, and other body parts with weakened muscles. Patients can sometimes be taught to use another set of muscles in place of muscles damaged by MD. A program of regular, light exercise can help keep muscles in good condition.

Surgery

Surgery may be necessary to correct some severe symptoms of MD. Contractures can be treated, for example, by cutting the damaged muscle. The muscle is then held in place until it grows back normally. In FSH, the shoulder blade can be braced to compensate for muscle weakness. For a person with OPMD, eyelids can be lifted by a surgical procedure to correct for drooping eyelids. Scoliosis can sometimes be corrected by back surgery. In this surgery, the vertebrae that make up the spine are fused (fixed) together. Steel rods are then inserted and attached to the vertebrae to keep the spine in a straight, stiff position.

Occupational Therapy

The purpose of occupational therapy is to help patients find ways of making up for their loss of strength and dexterity. Strategies may include changes in the home environment, learning to use special utensils and dressing aids, and use of a wheelchair and communication devices, such as hearing aids.

Nutrition

Good nutrition helps promote general health in all forms of MD. No special diet is needed or has been shown to relieve any of its symptoms, however.

Cardiac Care

EDMD and BMD may require certain kinds of treatment for heart problems. For example, drugs such as nifedipine (pronounced nie-FED-uh-peen) help maintain a regular heartbeat. An artificial pacemaker may also need to be installed in patients with an irregular heartbeat.

Respiratory Care

Weakness in diaphragm muscles can be a very serious condition that may result in a person losing the ability to breathe on his or her own. In such cases, a mechanical device may be needed to help the patient breathe. For example, air may be administered through a face mask or mouthpiece. Or a tracheotomy (pronounced TRAY-kee-OT-uh-mee) may be necessary. In a tracheotomy, a tube is inserted through a hole cut in the throat. Air can then be provided directly to the person's respiratory system.

Good lung hygiene is always necessary. Without proper care, infections of the lungs are common. Such infections can easily lead to pneumonia and even death. Patients with MD may also need to learn techniques for coughing. The normal cough reaction is usually difficult because of damaged muscles. But coughing is necessary to expel foreign particles that can cause disease and infection.

Experimental Treatments

Two experimental procedures may hold some promise for treating MD. One of these procedures is called myoblast transfer. In this procedure, millions of immature muscle cells are injected into a patient's damaged muscle. The goal of the procedure is to provide the person's body with normal, healthy cells that may be able to function in place of damaged ones. Thus far, there seems to be no evidence that this procedure is successful, although research continues in the hope of success in the future.

The second procedure is gene therapy. In gene therapy, a person with MD is injected with artificially produced genes that are correct copies of the faulty genes in their body. The hope is that the correct genes will function in cells the way the faulty genes are supposed to but don't. Gene therapy is a very difficult task complicated with many side effects. However, many researchers believe that it may be the most likely way of curing MD.

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