No treatment is currently available to stop or reverse any form of muscular dystrophy (MD). Instead, certain therapies and medications aim to treat the various problems that result from MD and improve the quality of life for patients. These include the following1:
Beginning physical therapy early can help keep muscles flexible and strong. A combination of physical activity and stretching exercises may be recommended.
Many people with MD do not realize they have little respiratory strength until they have difficulty coughing or an infection leads to pneumonia. Regular visits to a specialist early in the diagnosis of MD can help guide treatment before a respiratory problem occurs2. Eventually, many MD patients require assisted ventilation.
MD patients who experience weakness in the facial and throat muscles may benefit from learning to slow the pace of their speech by pausing more between breaths3 and by using special communication equipment.
As physical abilities change, occupational therapy can help patients with MD relearn these movements and abilities. Occupational therapy also teaches patients to use assistive devices such as wheelchairs and utensils.
At various times and depending on the form of MD, many patients require surgery to treat the conditions that result from MD. People with myotonic MD may need a pacemaker to treat heart problems or surgery to remove cataracts, a clouding of the lens of the eye that blocks light from entering the eye.
Certain medications can help slow or control the symptoms of MD. These include the following:
- Glucocorticoids, such as prednisone. Studies show that daily treatment with prednisone can increase muscle strength, ability, and respiratory function and slow the progression of weakness. Side effects may include weight gain.4 Long-term use may result in brittle bones, cataracts, and high blood pressure. The NIH’s Therapeutics for Rare and Neglected Diseases (TRND) Program is collaborating on a new glucocorticoid treatment called VBP15. Early clinical trial results show that the treatment may have the same positive results as prednisone, but without the side effects.5
- Anticonvulsants. Typically taken for epilepsy, these drugs may help control seizures and some muscle spasms.
- Immunosuppressants. Commonly given to treat autoimmune diseases such as lupus and eczema, immunosuppressant drugs may help delay some damage to dying muscle cells.
- Antibiotics to treat respiratory infections.
- National Institute of Neurological Disorders and Stroke (NINDS). (2011). NINDS muscular dystrophy information page. Retrieved May 25, 2012, from http://www.ninds.nih.gov/disorders/md/md.htm
- Moxley, R.T., III, Ashwal, S., Pandya, S., Connolly, A., Florence, J., Matthews, K., et al.; Quality Standards Subcommittee of the American College of Neurology; Practice Committee of the Child Neurology Society. (2005). Practice parameter: Corticosteroid treatment of Duchenne dystrophy. Neurology, 64, 13-20. Retrieved June 22, 2012, from http://www.neurology.org/content/64/1/13.full.pdf (PDF - 119 KB)
- Wahl, M. (2001, February). Sorting out speech services. Quest; 8(1). Retrieved June 22, 2012, from http://static.mda.org/publications/quest/q81speech.html
- American Thoracic Society Consensus Statement. (2004). Respiratory care of the patient with Duchenne muscular dystrophy. American Journal of Respiratory and Critical Care Medicine, 170, 456-465. Retrieved June 22, 2012, from http://ajrccm.atsjournals.org/content/170/4/456.full
- ReveraGen. (2011). ReveraGen BioPharma selected as awardee for inaugural National Institutes of Health’s Therapeutics for Rare and Neglected Diseases Program for its novel dissociative glucocorticoid analogue [press release]. Retrieved June 22, 2012, from http://www.duchennemd.org/assets/ReveraGen-TRND-Partnership-FINALpdf.pdf (PDF - 200 KB)