Positive updates for exon skipping and other treatments - View News Article - Action Duchenne - Fighting for a cure for muscular dystrophy

Positive updates for exon skipping and other treatments - View News Article - Action Duchenne - Fighting for a cure for muscular dystrophy: At a recent RNA and Oligonucleotide Therapeutics conference at Cold Spring Harbor, US, 10-13 April 2013, Dr John Kraus GSK Project Physician Leader presented Phase II results. The (DMD114117) study was an exploratory, unpowered, double blind, placebo-controlled clinical trial with the aim of assessing the safety and efficacy of drisapersen in 53 boys with DMD. The dosing regimens were continuous (6mg/kg/wk) and intermittent (10-week cycles of 9 doses at 6mg/kg over 6 wks, and 4 wks off drug).