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author：knowledge 2025-09-30 17:04:19 - browse：1

Sarepta - Cambridge -AP — Kristoffer Tripplaar/Sipa via AP

The Food and Drug Administration granted conditional approval Thursday to the first gene therapy for Duchenne muscular dystrophy. Regulators restricted the treatment to younger patients, with additional data required to broaden its use.

The gene therapy, called Elevydis, is made by Sarepta Therapeutics. The company will charge $3.2 million for the treatment, making it the U.S.’s second most expensive drug, behind a recently approved gene therapy for hemophilia. CEO Doug Ingram said on a conference call the price was below what a recently published company-funded analysis found would be cost-effective.

“The approval of Elevidys is a watershed moment for the treatment of Duchenne. Elevydis is the first and only gene therapy approved for Duchenne, and this approval brings us closer to our goal of bringing forward a treatment that provides the potential to alter the trajectory of this degenerative disease,” Ingram said.

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