The patient died of acute liver failure; the maker of the gene therapy noted that the patient also had a recent cytomegalovirus infection.

WVE-N531, an oligonucleotide, elicited significant functional benefit and reversal of muscle damage in the Phase II ...

Avidity Biosciences, Inc.'s innovative AOC platform and promising clinical programs make it a high-risk, high-reward ...

Wave Life Sciences’ experimental treatment for Duchenne muscular dystrophy hit the mark in a mid-stage study, setting the ...

Sarepta Therapeutics announced that a patient with Duchenne muscular dystrophy who received Elevidys (delandistrogene moxeparvovec-rokl) died following treatment.

NEW YORK, NY / ACCESS Newswire / March 24, 2025 / Bronstein, Gewirtz & Grossman, LLC is investigating potential claims on ...

Why didn’t analysts share the same fear that gripped investors about Sarepta stock? One reason is the known risk of acute liver injury associated with adeno-associated virus vector (AAV)-based gene ...

Things are going from bad to worse for Sarepta Therapeutics (NASDAQ: SRPT), a biotech focusing on gene therapies. The company ...

Sarepta Therapeutics (NASDAQ:SRPT – Free Report) had its target price lowered by Scotiabank from $105.00 to $80.00 in a ...

We gleaned this information from our observations today when Benzinga's options scanner highlighted 11 extraordinary options ...

By Deena Beasley (Reuters) -Gene therapy, with its offer of a possible cure for rare diseases like sickle cell, is losing ...

Sarepta Therapeutics (NASDAQ:SRPT – Get Free Report)‘s stock had its “sell” rating reiterated by analysts at HC Wainwright in ...