MedPage Today on MSN2d
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 ...
Managed Healthcare Executive1d
Promising Early Results of Investigational Duchenne Muscular Dystrophy Therapy
Avidity Biosciences Inc. says will submit an application for accelerated approval to the FDA for del-zota before the end of ...
BioSpace23h
In Wake of Elevidys Death, Duchenne Patient Community Vows to Push On
After Sarepta reported the death of a patient who had recently taken the gene therapy Elevidys, patient advocacy group Parent ...
Clinical Trials Arena on MSN1d
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 ...
Sportschosun (English) on MSN1d
New Treatment Strategy for Rare Disease Dutsenic Muscle Dystrophy...EZH2 inhibitor steroid effective in combination
Korean researchers have proposed a new treatment strategy that can increase the effectiveness of the genetic rare disease ...
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 ...
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 ...
BioPharma Dive3d
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 ...
HealthDay on MSN1d
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 ...
Becker's Hospital Review2d
1st patient death linked to muscular dystrophy treatment
Cambridge, Mass.-based Sarepta Therapeutics reported the first known death from its Duchenne muscular dystrophy gene therapy ...
The American Journal of Managed Care2d
EMBARK Data Show Continued Improvements With DMD Gene Therapy
Data from the EMBARK trial of delandistrogene moxeparvovec in patients with Duchenne muscular dystrophy (DMD) show that ...