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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, ...
No Accelerated FDA Approval For Edgewise's Rare Muscular Disease Drug; Shares Fall
Edgewise reports sustained functional stability in Becker patients and selects Phase 3 dose for Duchenne after positive Phase ...
MedPage Today on MSN1d
FDA Probes Gene Therapy After Two Deaths
The FDA is investigating the deaths of two boys with Duchenne muscular dystrophy who died soon after being treated with ...
Second patient death reported with gene therapy for muscular dystrophy
Shares of Sarepta Therapeutics have plunged again after the drugmaker reported a second death in connection with its gene ...
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 ...
Precision BioSciences Receives FDA Rare Pediatric Disease Designation for PBGENE-DMD for the Treatment of Duchenne Muscular Dystrophy
Precision BioSciences, Inc. (Nasdaq: DTIL), a clinical stage gene editing company utilizing its novel proprietary ARCUS® ...
Ticker: Second patient death reported with gene therapy for muscular dystrophy; Salmonella poisoning linked to pistachio cream
Ticker: Second patient death reported with gene therapy for muscular dystrophy; Salmonella poisoning linked to pistachio ...
STAT11d
Sarepta reports second patient death after treatment with Duchenne gene therapy
Sarepta said it was halting shipments of its Duchenne gene therapy for patients who can no longer walk, following the death ...
MedPage Today on MSN10d
Second Patient Dies After Gene Therapy for Duchenne Muscular Dystrophy
Duchenne muscular dystrophy patient died after being treated with the gene therapy delandistrogene moxeparvovec (Elevidys), ...
Biotech pauses trial after second patient death linked to gene therapy
Sarepta Therapeutics said a second patient had died after receiving its $3.2 million gene therapy to treat Duchenne muscular ...
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 ...
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 ...