The American Journal of Managed Care1d
Real-World Study Suggests DMD Gene Therapy Is Safe, Effective
Delandistrogene moxeparvovec, a gene therapy approved for the treatment of Duchenne muscular dystrophy (DMD), was found ...
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.
MedPage Today on MSN9d
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 ...
Clinical Trials Arena on MSN14h
Epicrispr secures $68m for facioscapulohumeral muscular dystrophy therapy trial
Epicrispr Biotechnologies has raised $68m in the initial close of its Series B financing round to commence the clinical trial ...
Gene therapy loses luster as investors eye quicker returns from weight-loss drugs
Gene therapy, with its offer of a possible cure for rare diseases like sickle cell, is losing early investors to ...
GEN - Genetic Engineering and Biotechnology News9d
DMD Patient Dies After Treatment with Sarepta Gene Therapy
The patient, whom Sarepta described as a young man and later told news outlets was 16 years old, suffered acute liver failure ...
BioPharma Dive9d
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 ...
MedCity News9d
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 ...
Gene-Therapy Maker Sarepta Tanks After Patient Death
Shares of Sarepta Therapeutics have struggled for the past year due to concerns about the commercial potential of its gene ...
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 ...