Clinical Trial ResultsA dose-dependent effect was not clearly visible in most of the functional endpoints including Stride Velocity 95th Centile, timed rise from floor, and timed 10-meter walk/run test at the 6 month mark.
Management ChangesChanges to the management team, including the stepping down of key executives, are expected to weigh on the stock.
Safety ConcernsThe tradeoff of magnitude of increased dystrophin expression vs the potential safety risk now appears less favorable.