Sarepta’s Gene Therapy Under Scrutiny After Patient Death
A patient has died while receiving Elevidys, a gene therapy for Duchenne muscular dystrophy, marking the first known death linked to the treatment. The company behind the drug, Sarepta Therapeutics, said that the patient suffered acute liver injury,
MedPage Today on MSN1d
Young Patient Dies After Receiving New Gene Therapy for Duchenne Muscular DystrophyA young person with Duchenne muscular dystrophy died following treatment with the recently approved gene therapy ...
Avidity Biosciences Inc. says will submit an application for accelerated approval to the FDA for del-zota before the end of ...
Cambridge, Mass.-based Sarepta Therapeutics reported the first known death from its Duchenne muscular dystrophy gene therapy ...
Regenxbio's RGX-202 gene therapy shows promising results in Duchenne muscular dystrophy, with high microdystrophin levels and ...
REGENXBIO (RGNX) reported new, positive interim data from two additional patients in the Phase I/II portion of the AFFINITY DUCHENNE trial of ...
Preservation of Skeletal Muscle Function Shown Over 3 Years Resulting in 52% Slowing of Disease-- --Data Presented at the ...
ROCKVILLE, MD, USA I March 19, 2025 I REGENXBIO Inc. (Nasdaq: RGNX) today reported new, positive interim data from two additional patients in the Phase ...
A Portland nonprofit is rewriting the story for people with disabilities by offering jobs, mentorship, and scholarships, ...
Data from the EMBARK trial of delandistrogene moxeparvovec in patients with Duchenne muscular dystrophy (DMD) show that ...
Results that may be inaccessible to you are currently showing.
Hide inaccessible results
Feedback