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FDA advisory panel narrowly approves Sarepta's Duchenne gene therapy for accelerated approval.

1 min read
Source: NPR
FDA advisory panel narrowly approves Sarepta's Duchenne gene therapy for accelerated approval.
Photo: NPR
TL;DR

FDA advisers have narrowly recommended approval of the first gene therapy for Duchenne muscular dystrophy, developed by Sarepta Therapeutics. The treatment involves a single infusion of viruses that has been genetically modified to carry a gene to patients' muscles to produce a miniature version of a protein called dystrophin. While the FDA is not bound by the recommendations of its outside advisers, it usually follows them. The agency is expected to decide by the end of May.

Topics:businesshealth#clinical-study#fda#gene-therapy#health#muscular-dystrophy#sarepta-therapeutics
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  1. FDA Duchenne muscular dystrophy gene therapy : Shots - Health News  NPR
  2. Tracking the FDA advisory panel on Sarepta's gene therapy  STAT
  3. US FDA advisers narrowly back Sarepta's Duchenne gene therapy for accelerated approval  Reuters
  4. FDA advisors narrowly vote in favor of Sarepta's DMD gene therapy  FierceBiotech
  5. For Sarepta and Muscular Dystrophy Patients, Today's FDA Hearing is Pivotal  Barron's

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