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 ...
MedPage Today on MSN2d
Shipments of Duchenne Gene Therapy to Resume After FDA Review of Patient Death
Sarepta's therapy has been under scrutiny from regulators after two teenage boys died earlier this year from acute liver ...
Medscape5d
EMA Says No to Duchenne Gene Therapy Elevidys
The European drug authority recommends refusal of marketing authorization after study shows no benefit over placebo.
Amazon S3 on MSN17h
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 ...
STAT6d
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 ...
FDA opens probe into mysterious death linked to gene therapy
The Food & Drug Administration is investigating the death of an 8‑year‑old following Elevidys gene therapy for Duchenne ...
STAT6d
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 ...
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 ...
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 ...
The Manila Times6d
Roche provides regulatory update on Elevidys™ gene therapy for Duchenne muscular dystrophy in the EU
EMA’s CHMP issued an opinion not to recommend Elevidys™ (delandistrogene moxeparvovec) for the treatment of ambulatory individuals with Duchenne muscular dystrophy (DMD) Roche will continue its ...
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.