Elevidys

article

Amid FDA turmoil Sarepta’s gene therapy worries begin to ease

1 August 2025 | By Dominic Tyer (European Pharmaceutical Review)

But the impact of George Tidmarsh’s CBER appointment after Vinay Prasad’s abrupt resignation remains to be seen.

news

Gene therapy approved for Duchenne muscular dystrophy

23 June 2023 | By Catherine Eckford (European Pharmaceutical Review)

The first approved gene therapy for certain paediatric Duchenne muscular dystrophy (DMD) patients leads to production of the Elevidys micro-dystrophin protein.

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Elevidys

Amid FDA turmoil Sarepta’s gene therapy worries begin to ease

Gene therapy approved for Duchenne muscular dystrophy