News

FDA Suspends Muscular Dystrophy Drug After 3 Patient Deaths
The U.S. Food and Drug Administration ( FDA) has placed an immediate clinical hold on Sarepta Therapeutics' investigational ...
Sarepta Therapeutics crisis is huge blow to Duchenne families, company
Sarepta rebuffed a call from the Food and Drug Administration to halt all shipments of its gene therapy for Duchenne muscular ...
Capricor Therapeutics Provides Regulatory Update on Deramiocel BLA for Duchenne Muscular Dystrophy
FDA issued Complete Response Letter Capricor plans to resubmit its BLA to include data from the ongoing Phase 3 HOPE-3 trial ...
STAT3dOpinion
We are mothers of Duchenne patients. Recent setbacks with Sarepta must not stop progress
As mothers of children with this disease, we have wept helplessly in recent months as friends — fellow members of a club we ...
Fierce Biotech3d
Sarepta LGMD trials all hit by FDA hold amid newly surfaced safety concerns over gene therapy
The Sarepta saga continues, with the FDA slapping a clinical hold across all of the company’s investigational limb-girdle ...
MedPage Today on MSN6d
Duchenne Gene Therapy Will Undergo Changes After Patient Deaths
At the FDA's request, delandistrogene moxeparvovec (Elevidys), the only approved gene therapy for Duchenne muscular dystrophy ...
Novel approach enhances neuromuscular function in patients with Duchenne muscular dystrophy
New research has identified the enzyme glutamate dehydrogenase 1 (GLUD1) as a new therapeutic target for Duchenne muscular ...
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 ...
STAT13d
FDA rejects Capricor’s cell therapy for Duchenne muscular dystrophy
The FDA rejected a Duchenne muscular dystrophy cell therapy from Capricor Therapeutics, as a larger study of the treatment ...
Sarepta drops as report suggests FDA needs new trials for Elevidys
Sarepta Therapeutics (SRPT) stock drops as the company faces potential FDA-mandated studies after safety concerns over its ...
pharmaphorum16d
Taiho's late-stage Duchenne candidate fails trial
Taiho Pharmaceutical has suffered a blow to its near-term pipeline after a drug candidate for Duchenne muscular dystrophy (DMD) missed the mark in a phase 3 trial.
Fierce Pharma2d
Sarepta, bowing to FDA pressure, pauses shipments of Duchenne gene therapy Elevidys
A brief skirmish between Sarepta Therapeutics and the FDA has ended before escalating into a full-on regulatory clash, as the company has bowed to the agency’s demand. | A brief skirmish between ...