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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.
Voelker R. First Corticosteroid Approved for Duchenne Muscular Dystrophy. JAMA. 2017;317(11):1110. doi:10.1001/jama.2017.1773
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