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