Duchenne Muscular Dystrophy Treatments: Deflazacort

Deflazacort (Emflaza) is a drug used in the treatment of Duchenne muscular dystrophy (DMD). It’s generally given to patients five years and older and is a type of corticosteroid precursor which acts as an immunosuppressant and anti-inflammatory medication.

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Conducted in 1995, clinical trials of the drug found that taking deflazacort over a prolonged period of time (52 weeks) resulted in improved muscle strength and in some cases basic functions also improved, like the length of time it took to stand, climb stairs or walk a certain distance.

Side effects of the drug included headaches, facial puffiness, increased appetite and weight gain (although less weight was gained by those on the trial compared to boys taking prednisone), cough and colds, increased urination during the day, and excessive hair growth.  Find out more about deflazacort here. 

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