UPDATED: Did Sarepta really score a success in the latest cut of their next-gen Duchenne MD drug? As usual, that's a little complicated
Sarepta boosters have been keeping their fingers crossed that the readout on their new-and-improved 30 mg/kg approach to exon-skipping for Duchenne muscular dystrophy patients will do much better than the lower dose they started out with. And this morning they were rewarded with a basic outline of success for the part A segment of their MOMENTUM study — though you can expect plenty of questions about its exploration with a tiny group of 4 patients, a continued reliance on a questionable biomarker with no actual proof of efficacy, evidence of serious toxicity with several adverse events and a curious case of predictive modeling to achieve the kind of success some analysts have been looking for.
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