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MedPage Today on MSN18h
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 ...
US FDA declines to approve Ultragenyx's gene therapy for rare genetic disorder
The U.S. Food and Drug Administration has declined to approve Ultragenyx Pharmaceutical's experimental gene therapy to treat ...
BioSpace2h
Sarepta Reports a Third Gene-Therapy Related Patient Death
The patient, who was being treated with an investigational gene therapy for limb-girdle muscular dystrophy, died of acute ...
BioPharma Dive20m
Patient reportedly dies in Sarepta study of limb-girdle gene therapy
According to multiple reports, a 51-year-old man with limb-girdle muscular dystrophy died from acute liver failure after ...
pharmaphorum2h
Third death reported with a Sarepta gene therapy
A third fatality has been recorded among patients receiving treatment with a Sarepta gene therapy, adding to the troubles ...
FierceBiotech4h
3rd patient dies after receiving a Sarepta gene therapy
The newest death reported was a 51-year-old man with limb-girdle muscular dystrophy (LGMD) type 2D/R3 who’d received Sarepta’s gene therapy candidate SRP-9004 in a phase 1 trial, Sarepta told ...
Sarepta Sinks After Another Patient Dies on Gene Therapy
Sarepta Therapeutics Inc. said another patient has died from acute liver failure after receiving one of its gene therapies, ...
Neurology Advisor56m
Child’s Rare Neurodegenerative Disease Fuels Therapy Innovation: Paxton’s Story
The Purdy family shares their insights on navigating their child’s rare neurodegenerative disease and their direct ...
Southwest Journal3h
Breaking: Second Patient Death Prompts FDA Investigation of Sarepta’s Elevidys Gene Therapy
Second patient death from liver failure after Sarepta's Elevidys gene therapy triggers FDA investigation. Stock crashes 41% ...
STAT7h
Patient dies in Sarepta gene therapy trial, adding to safety concerns
A 51-year-old man died last month after receiving an experimental treatment from Sarepta, the third death this year tied to ...
STAT1h
Pharmalittle: We’re reading about a GSK setback, another Sarepta gene therapy death, and more
In a surprise, a FDA advisory panel voted that risks tied to a GSK blood cancer drug called Blenrep outweighed the benefits ...
Sarepta plunges after third patient death tied to gene therapy - report
Sarepta Therapeutics (SRPT) said a third patient has died from acute liver failure after receiving one of its gene therapies, ...