Duchenne muscular dystrophy

News

Uncertainty ends Sarepta's Duchenne gene therapy Elevidys ends after an FDA label update, with the company's shares on the up despite restrictions.

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The ESSENCE trial of Sarepta's Vyondys 53 and Amondys 45 failed to hit its objective, placing their FDA accelerated approvals at risk.

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Novartis has made an offer to acquire RNA therapeutic specialist Avidity Biosciences, according to an unconfirmed report.

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Sarepta has restarted distribution of its Duchenne muscular dystrophy gene therapy Elevidys, but only for patients who can walk.

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The FDA seems to have won its staring contest with Sarepta over shipments of Elevidys, as the company blinks and agrees to a pause from today.

News

Sarepta has defied an FDA request to halt all use of its gene therapy for Duchenne muscular dystrophy, creating an intriguing regulatory impasse.