Teen’s death following Sarepta DMD gene therapy underscores a risk seen for decades
The death of a 16-year-old boy evokes a lengthy battle to realize the benefit of cutting-edge gene therapy while also ...
Hosted on MSN15d
Young Patient Dies After Receiving New Gene Therapy for Duchenne Muscular Dystrophy
In October 2023, Sarepta said the confirmatory EMBARK trial did not meet ... on the totality of the evidence" for delandistrogene moxeparvovec, and that several secondary endpoints in EMBARK and other ...
pharmaphorum2d
Despite trial miss, Sarepta plans new filing for DMD therapy
There were, however, significant improvements on secondary measures, including time to rise and a 10-metre walk test that, according to Sarepta, were “evidence of a clinically meaningful benefit ...
pharmaphorum6d
Sarepta bags key expansion to DMD gene therapy label in US
Sarepta’s gene therapy for Duchenne muscular ... In particular, it highlighted “compelling” improvements over placebo on secondary endpoints like improvements in time to rise from the ...
BioSpace14d
Sarepta’s Elevidys Death, FDA’s Vaccine Move, Weldon Whiplash, PDUFAs in Cardio
Sarepta will update Elevidys’ label after a patient died following treatment; the FDA issues flu vaccine recommendations ...
Seeking Alpha13d
Sarepta: Should Investors Run For The Exit?
Shares of Sarepta Therapeutics dropped 20% after a patient died from acute liver injury post-ELEVIDYS treatment, an event not previously reported. Despite the tragic news, we believe this is an ...
Seeking Alpha29d
Sarepta Therapeutics' Long-Term Potential Intact Despite Near-Term Stock Weakness
Sarepta Therapeutics' stock is at 52-week lows despite strong Q4 earnings and promising clinical updates, reflecting broader biotech sector challenges and investor sentiment. Q4 2024 earnings ...