The CureDuchenne site has also been posting notes from WMS 2014 for previous days. Click on link for additional info from earlier presentations. Lots of info.
Notes from Eteplirsen presentation:
Neutral charge as it passes through kidney; cleared through kidneyIV QWInclusion: >7 years to 13 years (original cohort 7-11 yr)ITT: 12 pts (4 each placebo, 30 & 50 mg/kg)2 pts stopped walking prior to 24 week time pointData at 144 weeks for modified ITT: 10 pts (6 pts eteplirsen and 4 placebo delay)At 144, delta 75 m (-107 vs –32)Both groups start producing dystrophin after 12 wk Tx, increasing over timeNo complete resolution, plateau and then slow decline; imperfect drugSlower decline in walking than natural historyPulmonary function test; remain stable (no stat diff)MIP and MEP stableNo AE at 144 wk; no liver enzymes, kidney fx, coagulation, etc.No ISRs, etc.Pooled the doses in the data; no diff between dose levels