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‘It’s a vote for hope’: first gene therapy for muscular dystrophy nears approval, but will it work?

The FDA’s decision, expected this month, follows several setbacks and delays and will pose difficult choices for the families of children with Duchenne muscular dystrophy.
  1. Sara Reardon
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Stacie Al-Chokhachi and her son who suffers from DMD, at a FDA meeting about a new drug treatment for the disease.

The parents of children with Duchenne muscular dystrophy have been pushing for swift approval of therapies. Credit: Eric Kruszewski/New York Times/Redux/eyevine

Update: The US Food and Drug Administration has now approved the first gene therapy for Duchenne muscular dystrophy for children aged four and five. The one-time treatment, named Elevidys, will cost US$3.2 million, according to Sarepta, the Cambridge, Massachusetts-based company that developed it.

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Nature 618, 451-453 (2023)

doi: https://doi.org/10.1038/d41586-023-01799-z

Updates & Corrections

  • Correction 02 June 2023: The US Food and Drug Administration told Sarepta that it would complete its review by 22 June, not 27 June as previously reported.

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