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News From the Food and Drug Administration
March 21, 2017

First Corticosteroid Approved for Duchenne Muscular Dystrophy

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Copyright 2017 American Medical Association. All Rights Reserved. Applicable FARS/DFARS Restrictions Apply to Government Use.

JAMA. 2017;317(11):1110. doi:10.1001/jama.2017.1773

Deflazacort has become the first corticosteroid to receive FDA approval to treat patients aged 5 years and older who have Duchenne muscular dystrophy.

The drug’s effectiveness was shown in a study of 196 male patients aged 5 to 15 years with a documented dystrophin gene mutation and muscle weakness before they were 5 years old. After 12 weeks, patients who took deflazacort had clinically improved muscle strength compared with those who received a placebo. Muscle strength remained stable among patients treated with deflazacort through the end of the 52-week study. In another study with 29 male patients, those who received the drug also had muscle strength improvements compared with the placebo group.

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