Cantor Fitzgerald reiterated their overweight rating on shares of Sarepta Therapeutics (NASDAQ:SRPT – Free Report) in a ...

Cambridge-based Sarepta Therapeutics said Tuesday that a patient died while taking its closely watched gene therapy for ...

A young person with Duchenne muscular dystrophy died following treatment with the recently approved gene therapy ...

Sarepta Therapeutics said on Tuesday a young man has died due to acute liver failure after treatment with its gene therapy for a rare muscular dystrophy.

The company plans to submit a biologics licence application to the US Food and Drug Administration later this year.

After some earlier data sparked questions from analysts, Avidity Biosciences has released more data for its RNA-based ...

Dyne is eyeing an accelerated approval filing for DYNE-251 in early 2026 that would pit the asset against Sarepta’s Exondys ...

The Phase I/II Deliver trial sought to establish the impact of DYNE-251 on several mobility-related endpoints, seeing an ...

Italfarmaco S.p.A. today announced a comprehensive update on the regulatory and clinical advancements for givinostat, the company’s drug for the treat ...

This will allow us to gather additional safety data, assess the impact of this dose of PGN-EDO51 on dystrophin levels, and potentially improve the design of CONNECT2,” said James McArthur ...

63% of mice treated with two rAAV9-microdystrophin doses produced antibodies to dystrophin, which was less in mice treated with two rAAV9-microdystrophin doses and combination therapy (25%). Likewise, ...

Entrada Therapeutics can plough on with the development of its Duchenne muscular dystrophy (DMD) therapy, ENTR-601-44, now that the US Food and Drug Administration (FDA) has lifted a two-year ...