Experts review current and potential treatments for Duchenne muscular dystrophy | DuchenneXchange

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Experts review current and potential treatments for Duchenne muscular dystrophy


Experts provided an overview of the many approaches to counteract the loss of muscle mass, inflammation, fibrosis, calcium overload, oxidative stress, and ischemia (inadequate blood supply) in Duchenne muscular dystrophy (DMD), without actually targeting the cause, a mutation in the dystrophin gene.

The review, “Pharmacological therapeutics targeting the secondary defects and downstream pathology of Duchenne muscular dystrophy,” was published in the journal Expert Opinion on Orphan Drugs.

The review notes that the corticosteroids prednisone and prednisolone, often used to treat DMD, have unwanted side effects, while deflazacort, approved this year by the U.S. Food and Drug Administration (FDA) for DMD patients older than 5, has shown to cause less weight gain than the other drugs.

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