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Neuromuscular Disease and Spinal Muscular Atrophy | Overview


Duchenne muscular dystrophy: double-blind randomized trial to find optimum steroid regimen (FOR-DMD)

Steroid therapy is now a standard treatment for children affected by Duchenne Muscular Dystrophy (DMD). The FOR-DMD study will compare three ways of giving steroid treatment to boys with DMD to determine which of the three ways increases muscle strength the most, and which causes the fewest side effects. The steroids that will be used in this study are prednisone and deflazacort. The results of this study will have a direct impact on the current and future management of boys with DMD throughout the world.

Sarepta PROMOVI 4658-301- An Open-Label, Multi-Center, 48-Week Study with a Concurrent Untreated Control Arm to Evaluate the Efficacy and Safety of Eteplirsen in Duchenne Muscular Dystrophy 

The purpose of this research study is to evaluate the efficacy and safety of eteplirsen in patients with Duchenne Muscular Dystrophy (DMD). This study will test whether eteplirsen works to improve muscle function in boys with DMD who have genetic deletions that may be corrected by exon-skipping the gene called exon 51. Eligible participants are males diagnosed with DMD between 7 to 16 years of age who are currently taking corticosteroids. Participants must also be able to walk a distance of greater than or equal to 300m during the study’s Screening period.