Background: On Monday, June 29, BioMarin announced that the FDA has accepted their New Drug Application (NDA) for drisapersen and Sarepta announced that they have completed their NDA submission for eteplirsen.  Both “exon-skipping” drugs target exon 51, and may help up to 13% of Duchenne muscular dystrophy patients. Exon skipping is a treatment strategy in which sections of genetic . . .

Press Release: Akashi Therapeutics Reports Positive Clinical Data on HT-100 in Patients with Duchenne Muscular Dystrophy Statement from MDA Scientific Program Officer Laura Hagerty, Ph.D.: “Data from this ongoing clinical trial shows that boys with DMD who were treated with HT-100 demonstrated improvements in muscle strength. If these positive trial results are borne out in future . . .

Background:  On June 9th, the U.S. Food and Drug Administration (FDA) released a draft containing guidance to assist drug companies working to develop drugs for the treatment of Duchenne muscular dystrophy (DMD) and related diseases including Becker muscular dystrophy, DMD-associated cardiomyopathy and symptomatic DMD carrier status in females. The guidance relates FDA perspective on a number . . .

Press Release: BioMarin Completes Rolling NDA Submission to FDA for Drisapersen for Treatment of Duchenne Muscular Dystrophy Amenable to Exon 51 Skipping Statement from MDA Scientific Program Officer Laura Hagerty, Ph.D.: “This is very encouraging news for the DMD community, and we hope it means that a treatment is on the horizon. If drisapersen were to . . .

Press Release: Santhera’s Positive Phase III Trial (DELOS) in Patients with Duchenne Muscular Dystrophy Published in The Lancet “This is welcome news for our community,” said MDA Senior Vice President and Scientific Program Director Grace Pavlath, Ph.D. “The need for effective DMD therapies is urgent, and we look forward to the possibility that idebenone may . . .

Press Release: Santhera receives FDA Fast Track Designation for Raxone®/Catena® (idebenone) for theTreatment of Duchenne Muscular Dystrophy Statement from MDA Senior Vice President and Scientific Program Director Grace Pavlath, Ph.D.: “This is exciting news for our community. MDA is committed to bringing safe and effective treatments and cures to kids and adults living with neuromuscular . . .