Duchenne muscular dystrophy
- Duchenne and Becker muscular dystrophy
- Muscular dystrophy, Duchenne
- Muscular dystrophy, pseudohypertrophic progressive, Duchenne type
- Severe dystrophinopathy, Duchenne and Becker type
Your QuestionIs there treatment available to cure Duchenne muscular dystrophy or slow the progression of symptoms?
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Questions on this page
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.
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:
- 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.
Exon skipping drugs are currently being researched as a treatment option for DMD. This treatment is designed to get the muscle cells of individuals with Duchenne muscular dystrophy to produce the missing protein, called dystrophin. Many different pharmaceutical companies (GlaxoSmithKline, PTC Therapeutics, and AVIBioPharma) are working on developing different versions of this treatment. The different names that have been used for these drugs are PRO051 (GSK2402968), PTC124 (ataluren), and eteplirsen. The type of mutation an individual has will determine if an individual is eligible for this exon skipping treatment. This type of treatment is expected to be approved in 2014.
Another type of treatment is Coenzyme Q10, which is an antioxidant that can be used in addition to prednisone (a steroid) to help improve the symptoms of DMD. Multiple studies have shown an increase in muscle strength when using this treatment.
Also, a small study was done to look at the effectiveness of the use of idebenone therapy in children. This treatment was safe and well tolerated and seemed to help improve the symptoms of DMD.
There is also another treatment that will soon be studied in clinical trials. This treatment combines three medicines, sildenafil, spironolactone, and ibuprofen. This combination of medicines was found to reduce the severity of DMD in mice.
Two other treatment options for DMD have been studied but neither of these has improved symptoms. One treatment is called pentoxifylline, which helps improve circulation of blood in the body. The other treatment is with glutamine, which is an amino acid essential for muscle strength. Further research may be done to find out ways to make these treatments effective.
The DuchenneConnect Profile provides a patient registry for individuals with Duchenne muscular dystrophy. This resource gives people access to information about new treatments and trials, services like genetic testing and counseling, and regional and local resources for patients and their families.
In addition, the Muscular Dystrophy Association (MDA) MDA offers quality medical care from doctors, nurses and therapists experienced in dealing with neuromuscular diseases at 225 hospital-affiliated clinics. These clinics also serve as sites for clinical trials of the latest experimental therapies and drugs. You can contact the MDA directly for more information.
Muscular Dystrophy Association (MDA) - USA
3300 E. Sunrise Drive
Tucson, AZ 85718
Web site: http://www.mdausa.org
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- Buyse GM, Goemans N, van den Hauwe M, et.al. Idebenone as a novel, therapeutic approach for Duchenne muscular dystrophy: results from a 12 month, double-blind, randomized placebo-controlled study. Neuromuscular Disorders. 2011 Jun; 21(6):396-405. http://www.ncbi.nlm.nih.gov/pubmed/21435876. Accessed 11/4/2013.
- Escolar DM, Zimmerman A, Bertorini T, et al. Pentoxyifylline as a rescue treatment for DMD: a randomized double-blind clinical trial. Neurology. 2012 Mar; 78(12):904-13. http://www.ncbi.nlm.nih.gov/pubmed/22402864. Accessed 11/4/2013.
- Xi P, Jiang Z, Zheng C, Lin Y, Wu G. Regulation of protein metabolism by glutamine: implications for nutrition and health. Frontiers in Bioscience. 2011 Jan; 16:578-97. http://www.ncbi.nlm.nih.gov/pubmed/21196190. Accessed 11/4/2013.
- Mok E, Letellier G, Cuisset JM, et al. Lack of functional benefit with glutamine versus placebo in Duchenne muscular dystrophy: a randomized crossover trial. PLoS One. 2009 May; 4(5):e5448. http://www.ncbi.nlm.nih.gov/pubmed/19421321. Accessed 11/4/2013.