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Duchenne muscular dystrophy (DMD)

thunderstorm road
Pharma

Patient dies after receiving Sarepta's DMD gene therapy Elevidys

The incident brings attention to the safety of AAV-based gene therapies and could spark renewed concerns over the benefit-risk profile of Elevidys.
Angus Liu Mar 18, 2025 10:34am
The Top Line hero image Valentines Day hearts

A heartwarming review of cardiovascular news, most-loved stories

Feb 14, 2025 9:00am
Sarepta Sarepta Therapeutics

Sarepta's Elevidys blows sales expectations out of the water

Nov 7, 2024 3:43pm
Duchenne muscular dystrophy DMD

FDA broadens label for Sarepta's Duchenne gene therapy Elevidys

Jun 21, 2024 7:51am
stop light green

Italfarmaco wins FDA's blessing for nonsteroidal DMD therapy

Mar 22, 2024 11:05am
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PTC, after rejections, will try again with Translarna at the FDA

Mar 19, 2024 10:10am

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