Monday 24 June 2013

Updates in exon skipping data - View News Article - Action Duchenne - Fighting for a cure for muscular dystrophy

Updates in exon skipping data - View News Article - Action Duchenne - Fighting for a cure for muscular dystrophy: Sarepta Therapeutics, Inc. announced updated data at the Wells Fargo Securities Healthcare Conference on the 19th of June from Study 202, a Phase IIb open-label extension study of eteplirsen for patients who have a genotype amenable to skipping of exon 51. Results at 84 weeks showed a continued stabilisation of walking ability in eteplirsen-treated patients evaluable on the 6-minute walk test (6MWT).As previously reported, increased novel dystrophin was observed as assessed by muscle biopsy at week 48 and is now in the long-term extension phase in which patients continue to be followed for safety and clinical outcomes. 

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