Is Sarepta’s Gene Therapy Elevidys Casting Doubt On A Popular DMD Endpoint?

Sponsors don’t seem worried that US FDA will become skeptical of the North Star Ambulatory Assessment (NSAA) because Sarepta says it was not sensitive enough to detect patient changes in the confirmatory trial for its DMD gene therapy Elevidys.

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Will sponsors have to adjust the NSAA after Sarepta said the scale was not sensitive enough to detect patient changes? • Source: Shutterstock

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