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Sarepta’s DMD Gene Therapy, Like Exondys 51, Is Foundational, Advocates Argue

 
• By Derrick Gingery

The Duchenne muscular dystrophy candidate should be approved because it will help grow development in the space, patient groups argue, not just because of the value the gene therapy could provide to those receiving the treatment itself.

foundation
An approval of SRP-9001 will help other gene therapy improvements move forward, CureDuchenne's chief scientific officer said. • Source: Shutterstock

Just as they did with Sarepta Therapeutics, Inc.’s Exondys 51 (eteplirsen), patient groups view the company’s gene therapy candidate in Duchenne muscular dystrophy as a foundation to unlock more investment in the disease.

SRP-9001 (delandistrogene moxeparvovec), which is under priority review for accelerated approval by the US Food and Drug Administration, will benefit patients, but improvements already are in development, said

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