Beyond Exon 51 - PPMD Community

Beyond Exon 51 - PPMD Community: As Sarepta (formely AVI) announces promising results from its phase II interim data�and GSK follows up its promising phase II data with a phase III study, things are looking good for an exon-skipping based therapy for Duchenne…if you happen to be in the 13% of those with Duchenne who have a mutation that can be improved by skipping exon 51. For quick reference, this group includes out-of-frame deletions in exons 45-50, 47-50, 48-50, 49-50, 50, and 52.