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 ...
BioSpace4d
Sarepta Tags DMD Gene Therapy Elevidys With Black Box Warning, Axes 500 Staff
Following the death of two teenage patients with Duchenne muscular dystrophy following Elevidys treatment, Sarepta ...
STAT2dOpinion
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 ...
Muscular Dystrophy News3d
Dosing begins in gene therapy trial for DMD-linked heart disease
The first patient has been dosed in a clinical trial testing Sardocor's one-time gene therapy for cardiomyopathy associated ...
News-Medical.Net on MSN6d
New cellular entry pathway improves gene therapy outcomes
Scientists from the Centenary Institute and the University of Sydney have made a landmark discovery that could lead to safer ...
STAT4d
Sarepta Therapeutics lays off one-third of workforce in drastic cost-cutting move
Sarepta Therapeutics announced it has laid off more than one-third of its workforce, a drastic cost-cutting move following ...
Fierce Pharma6d
Ultragenyx takes another hit as FDA snubs AAV gene therapy over manufacturing issues
The manufacturing-related rejection for UX111 is more than a setback for Ultragenyx, as it also delays a key test of the new ...