News

Parents call on FDA for path forward after Duchenne's gene therapy pause | Morning in America
The Food and Drug Administration has paused distribution of the gene therapy Elevidys following the death of a third patient ...
Amazon S3 on MSN7h
The story of Sarepta's Duchenne gene therapy
The last couple of weeks has been particularly distressing for Duchenne muscular dystrophy patients and their families. The ...
Chemistry World12h
Sarepta gene therapy deaths highlight tragic rare disease dilemma
Regulators block Duchenne muscular dystrophy treatment after fatal side-effects outweigh questionable efficacy ...
Just The News23h
FDA allows distribution of muscular dystrophy drug again after public criticism
The agency’s now-reversed decision to halt distribution of a gene therapy for children suffering from muscular dystrophy ...
Fierce Pharma2d
FDA takes U-turn on Sarepta's Elevidys, backing Duchenne gene therapy again in ambulatory patients
Monday, the FDA said Sarepta may resume treating ambulatory DMD patients with Elevidys. The recommendation comes just 10 days ...
FDA asks Sarepta to stop shipping Duchenne gene therapy
The company refused the FDA’s request and will continue shipping its therapy, Elevidys, to Duchenne patients who can still ...
STAT5d
For many Duchenne families, halt to gene therapy is heartbreak upon heartbreak
As gene therapy maker Sarepta Therapeutics tangles with FDA over its Duchenne treatment, patients and their families are ...
Sarepta will resume gene therapy shipments after FDA review of recent patient death
Shares of beleaguered drugmaker Sarepta Therapeutics jumped in afterhours trading Monday after the company said it would ...
FDA Lifts Safety Hold On Sarepta's Muscular Dystrophy Gene Therapy—Wall Street Cheers
Sarepta stock sees upgrades and price hikes as analysts reassess Elevidys prospects following a favorable FDA development.
STAT5d
Europe moves to reject embattled Duchenne muscular dystrophy gene therapy
European regulators said that a Duchenne gene therapy should not be approved, imperiling the treatment that is also facing ...
Yahoo Finance2y
FDA Approves First Gene Therapy for Duchenne Muscular Dystrophy
A new pioneering gene therapy Elevidys works by replacing the Duchenne variant of dystrophin with man-made version based on a version found in a patient with a milder form of the disease, called ...
The Globe and Mail14y
Pfizer’s Gene Therapy Study for Duchenne Muscular Dystrophy: A ...
The study aims to evaluate the safety and efficacy of a gene therapy, PF-06939926, in boys with Duchenne Muscular Dystrophy (DMD), a severe genetic disorder characterized by progressive muscle ...