News

FDA probes deaths of boys taking Sarepta Duchenne drug
The FDA said yesterday that it is investigating the deaths from liver failure of two non-ambulatory boys with Duchenne ...
Fierce Pharma1d
FDA investigates patient deaths after treatment with Sarepta's Duchenne gene therapy Elevidys
In one of the first major tests of the new FDA leadership’s regulatory philosophy toward gene therapies for rare diseases, ...
STAT8dOpinion
Following second patient death, Duchenne muscular dystrophy families deserve answers about Elevidys
Duchenne muscular dystrophy families are sharing stories about Elevidys complications on social media. But they aren’t ...
FDA investigates patient deaths after treatment with Sarepta's gene therapy
The U.S. Food and Drug Administration said on Tuesday that it is investigating reports of two deaths due to acute liver ...
STAT10d
After second Sarepta death, Duchenne muscular dystrophy community is racked by recrimination and worry
Another gene therapy death of a Duchenne patient has some parents and doctors criticizing the FDA's actions, even as other ...
BioSpace1d
FDA Reviews Sarepta’s Elevidys After Second Patient Death
The FDA is assessing the need for “further regulatory action” on Sarepta's Duchenne muscular dystrophy gene therapy in the ...
BioPharma Dive10d
Second Duchenne patient dies after receiving Sarepta gene therapy
Another person treated with Elevidys suffered acute liver failure, leading Sarepta to discontinue treatment in certain ...
Second patient death reported with gene therapy for muscular dystrophy
WASHINGTON (AP) — Shares of Sarepta Therapeutics plunged Monday after the biotech drugmaker reported a second death in ...
Sarepta reports second patient death after treatment with Duchenne gene therapy
Cambridge-based Sarepta Therapeutics said it was halting shipments of its Duchenne muscular dystrophy gene therapy for ...
Business Wire11d
Sarepta Provides Safety Update for ELEVIDYS and Initiates Steps to Strengthen Safety in Non-Ambulatory Individuals with Duchenne
Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, today provided a safety ...
MedCity News10d
Sarepta Stops Stop Shipments of Muscular Dystrophy Gene Therapy After Second Patient Death
A patient has died after receiving Sarepta Therapeutics’ approved gene therapy for Duchenne muscular dystrophy — the second fatality reported by the company in the past three months. Like the ...
MedPage Today on MSN10d
Second Patient Dies After Gene Therapy for Duchenne Muscular Dystrophy
To date, over 900 Duchenne patients have been treated with delandistrogene moxeparvovec. "The signal for ALF is exceptionally ...