MedPage Today on MSN7d
Young Patient Dies After Receiving New Gene Therapy for Duchenne Muscular Dystrophy
A young person with Duchenne muscular dystrophy died following treatment with the recently approved gene therapy ...
Patient dies following muscular dystrophy gene therapy, Sarepta reports
A patient has died while taking a closely watched gene therapy for muscular dystrophy. Sarepta Therapeutics announced the ...
Teenager who received Elevidys, Sarepta’s Duchenne gene therapy, dies
Elevidys was approved after intense debate at the FDA, as officials and reviewers disagreed over how strong the evidence was ...
Rheumatology Advisor5h
Patient Dies Following Treatment With DMD Gene Therapy Elevidys
Sarepta Therapeutics announced that a patient with Duchenne muscular dystrophy who received Elevidys (delandistrogene moxeparvovec-rokl) died following treatment.
BioPharma Dive7d
Duchenne patient dies after receiving Sarepta gene therapy
A young man treated with Elevidys died of acute liver failure. The case may give doctors pause before prescribing the ...
Northern Minnesota boy is first American to receive groundbreaking treatment for muscular dystrophy
An 8-year-old northern Minnesota boy is one of the first in the country to receive a specific type of gene therapy treatment.
MedCity News7d
Liver Failure-Associated Death Reported in Patient Treated With Sarepta Gene Therapy Elevidys
A patient has died following treatment with Elevidys, the Sarepta Therapeutics product that is the only FDA-approved gene therapy for Duchenne muscular dystrophy, the company disclosed Tuesday.
Regenxbio Touts Positive Biomarker Data From Duchenne Muscular Dystrophy Gene Therapy Trial
Regenxbio's RGX-202 gene therapy shows promising results in Duchenne muscular dystrophy, with high microdystrophin levels and ...
Clinical Trials Arena on MSN5d
Regenxbio’s DMD gene therapy drives 122.3% biomarker uptick in three-year-old
The interim data found one child in the Duchenne trial produced more than double the amount of the protein needed to maintain ...
Sarepta’s Gene Therapy Under Scrutiny After Patient Death
A patient died from liver injury while taking the gene therapy Elevidys Sarepta says more than 800 patients have received the ...
U.S. News & World Report7d
Patient Dies Following Muscular Dystrophy Gene Therapy, Sarepta Reports
It’s the first known patient death with the therapy, which has been ... about its effectiveness in treating Duchenne muscular dystrophy. It’s the first gene therapy approved in the U.S ...