03 November 2025

Rinse, Repeat

Sarepta has been a perpetual pinata for both skeptics and shorts from the beginning of its attempts to make an effective duchenne muscular dystrophy medicine. So far, it's not yet shown a clinical trial win in either PIII or post-approval confirmatory trials. But, if you bought up some AVII (what it started out as) when they started down this path, you'd have netted quite a bundle. Not today, though. Here's some from the latest confirmatory trial
Topline results found that numerical trends favored treatment versus placebo; however, the observed difference of 0.05 steps/second in least square means (LSM), did not reach statistical significance (P=0.309) on the primary endpoint, the 4-step ascend velocity at 96 weeks.
In case you're unfamiliar, that p-value bites pig's feet.

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