New evidence pushes Duchenne Muscular Dystrophy drug potentially closer to approval

Written by Alice Weatherston

Rare disease specialists, Marathon Pharmaceuticals, have announced conclusions from a Phase III study of the investigational glucocorticoid, termed deflazacort, for the treatment of Duchenne Muscular Dystrophy (DMD). The results were presented at the American Academy of Neurology (AAN) 68th Annual Meeting (15 – 21st April, Vancouver, BC, Canada). The trial was a randomized, double-blind placebo controlled and active comparator study that involved 196 patients. All patients received either deflazacort 0.9 mg/kg/day, deflazacort 1.2 mg/kg/day, prednisone 0.75 mg/kg/day or placebo for 12 weeks. Previously presented primary endpoints of the study indicated that deflazacort improved muscle strength in DMD patients in comparison...

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