Duchenne and becker muscular dystrophy


Is there an FDA approved treatment for Duchenne muscular dystrophy?

There is no cure for Duchenne muscular dystrophy (DMD). There are, however, several treatments used to help reduce the health problems seen in DMD such as physical therapy, exercise, and steroids. Physical therapy, braces, and active/passive exercise is done to build muscle strength and prevent a shortening, hardening, and stiffness of joints (contractures). Corticosteroids like prednisone and deflazacort are used to slow the progression of muscle weakness in people with DMD. There are several treatments being tried in research studies that could work to address the underlying problem in DMD. One option to learn more about possible treatment plans is to visit the "Standards of Care" webpage at CureDuchenne's website.

In September of 2016, the U.S. Food and Drug Administration (FDA) approved an weekly infused medication called EXONDYS 51™ (eteplirsen) made by Sarepta Therapeutics for about 13% of patients with Duchenne and Becker muscular dystrophy (DMD/BMD) who have a specific category of "misspelling" or gene mutation. This treatment will not work for every patient with DMD/BMD, just those with nonsense mutations that will allow skipping a part of the gene called exon 51. In order to learn if this medication is an option, you can speak with your main DMD/BMD doctor. If you do not have a DMD/BMD expert team who works with you, support groups such as Parent Project Muscular Dystrophy have information on their websites to help you find expert doctors at Certified Duchenne Care Programs with experience working with patients affected by DMD/BMD.

SOURCE: Emory University - Department of Human Genetics in collaboration with ThinkGenetic • https://www.thinkgenetic.com/diseases/duchenne-and-becker-muscular-dystrophy/treatment/7801 • DATE UPDATED: 2016-09-25


NORD RARE Disease summary on DMDhttp://rarediseases.org/rare-diseases/duchenne-muscular-dystrophy/

Darras BT, Miller DT, Urion DK. Dystrophinopathies. GeneReviews website. http://www.ncbi.nlm.nih.gov/books/NBK1119/

Sarepta Website information on pipeline. Sarepta Pipeline

MDA Informational page on eteplirsen

Sarepta Press Release on FDA approval

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