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

 
• By Derrick Gingery

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.

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

Sarepta Therapeutics, Inc. may have unearthed new questions about a popular Duchenne muscular dystrophy endpoint during its development of the gene therapy Elevidys, but they do not appear to be cascading across gene therapy development in the rare disease.

Elevidys (delandistrogene moxeparvovec-rokl) is the first of several highly anticipated gene therapies intended to treat DMD

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