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Diseases

Genetic and Rare Diseases Information Center (GARD)

Duchenne muscular dystrophy


Other Names for this Disease
  • Muscular dystrophy, Duchenne
  • DMD
  • Muscular dystrophy, pseudohypertrophic progressive, Duchenne type
See Disclaimer regarding information on this site. Some links on this page may take you to organizations outside of the National Institutes of Health.

Treatment

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How might Duchenne muscular dystrophy be treated?

There is no known cure for Duchenne muscular dystrophy (DMD). Treatment is aimed at the control of symptoms to maximize the quality of life.[1] Individuals with DMD often experience dilated cardiomyopathy (the heart becomes larger and weaker). This can be treated with medications and in severe cases a heart transplant may be necessary. Assistive devices for breathing difficulties may be needed, especially at night.[2]

 

Physical activity is encouraged for individuals with Duchenne muscular dystrophy. Physical inactivity (such as bed rest) can worsen the muscle disease. Physical therapy may be helpful to maintain muscle strength and function. Orthopedic devices (such as braces and wheelchairs) may improve the ability to move and take care of oneself.[1]

 

Steroids are usually given to individuals with Duchenne muscular dystrophy to help improve the strength and function of muscles. There are a few different steroids that can be used to treat DMD:[2]

 

  • Prednisone is a steroid that has been shown to extend the ability to walk by 2 to 5 years. However, the possible side effects of prednisone include weight gain, high blood pressure, behavior changes, and delayed growth.
  • Deflazacort (another form of prednisone), is used in Europe and believed to have fewer side effects.
  • Oxandrolone, a medication used in a research study, also has similar benefits to prednisone, but with fewer side effects.

Cyclosporine
has also been used as a treatment for DMD, and has improved muscle function in children. Although, its use is controversial because it can cause myopathy, which is a muscle disease that causes muscle weakness.

 

There are several other therapies that are also being researched, including exon skipping drugs, coenzyme Q10, idebenone, glutamine, and pentoxifylline.[2]
Last updated: 11/4/2013

References
  1. Haldeman-Englert C. Duchenne muscular dystrophy. MedlinePlus. February 3, 2014; http://www.nlm.nih.gov/medlineplus/ency/article/000705.htm. Accessed 4/25/2015.
  2. Learning About Duchenne Muscular Dystrophy. National Human Genome Research Institute (NHGRI). April 18, 2013; http://www.genome.gov/19518854. Accessed 4/25/2015.


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Management Guidelines

  • Section on Cardiology and Cardiac Surgery. Cardiovascular health supervision for individuals affected by Duchenne or Becker Muscular Dystrophy. Pediatrics 2006; 116: 1569-1573.
  • GeneReviews provides current, expert-authored, peer-reviewed, full-text articles describing the application of genetic testing to the diagnosis, management, and genetic counseling of patients with specific inherited conditions.
  • Orphanet Emergency Guidelines is an article which is expert-authored and peer-reviewed that is intended to guide health care professionals in emergency situations involving this condition.  
  • Project OrphanAnesthesia is a project whose aim is to create peer-reviewed, readily accessible guidelines for patients with rare diseases and for the anesthesiologists caring for them. The project is a collaborative effort of the German Society of Anesthesiology and Intensive Care, Orphanet, the European Society of Pediatric Anesthesia, anesthetists and rare disease experts with the aim to contribute to patient safety.

Clinical Trials & Research for this Disease

  • ClinicalTrials.gov lists trials that are studying or have studied Duchenne muscular dystrophy. Click on the link to go to ClinicalTrials.gov to read descriptions of these studies.
  • Orphanet lists European clinical trials, research studies, and patient registries enrolling people with this condition. 
Other Names for this Disease
  • Muscular dystrophy, Duchenne
  • DMD
  • Muscular dystrophy, pseudohypertrophic progressive, Duchenne type
See Disclaimer regarding information on this site. Some links on this page may take you to organizations outside of the National Institutes of Health.