Shares of Sarepta Therapeutics SRPT plunged nearly 24% last week after the company reported the death of a patient following treatment with Elevidys, its one-shot gene therapy for Duchenne ...

The leading gene therapy for DMD is Sarepta Therapeutics' (SRPT) Elevidys, approved by the FDA in June 2023 for ambulatory pediatric patients aged 4 to 5 with a confirmed DMD mutation. It delivers ...

The death of a patient treated with Sarepta's Duchenne muscular dystrophy (DMD) gene therapy Elevidys has played havoc with its share price – although analysts believe that its benefits still ...

Sanofi SA gained U.S. FDA approval for fitusiran as a first-in-class siRNA therapy for hemophilia. Branded Qfitlia, the antithrombin-lowering therapy is indicated for use as a prophylactic treatment ...

Digital therapeutics coupled with drugs has the potential to transform health care and change the way patients are treated, Anthony Costello, CEO of Medidata Solutions Inc., told BioWorld. Medidata ...

The sector was also shaken by the death of a patient who was dosed with Sarepta Therapeutic’s DMD gene therapy Elevidys (delandistrogene moxeparvovec), after experiencing acute liver failure.

which Leerink Partners’ Joseph Schwartz noted was greater than Sarepta Therapeutics showed with its Elevidys gene therapy, which the FDA approved in 2023.

which Leerink Partners’ Joseph Schwartz noted was greater than Sarepta Therapeutics showed with its Elevidys gene therapy, which the FDA approved in 2023. But perhaps more notably, Wave touted ...

At present, the market for DMD therapies is largely driven by Sarepta Therapeutic’s Elevidys (delandistrogene moxeparvovec) and Santhera Pharmaceuticals’ Agamree (vamorolone), with research by ...

As it gears up to submit an approval application next year, Wave Life Sciences has presented fresh phase 2 data showing its Duchenne muscular dystrophy (DMD) therapy is up to the task. The latest ...