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Sarepta shares plunge 30% as future of its gene therapy appears at risk
Shares of Sarepta Therapeutics plunged more than 30% on Friday as the future of its approved gene therapy appeared at risk.
Gene therapy developer Sarepta faces third patient death
Investors showed growing mistrust of the Sarepta team, repeatedly asking why the Cambridge company had not disclosed the ...
The Union Democrat1h
Rescued by fat bubbles: Scientists treat rare genetic disease with designer molecule
Tiny fat bubbles carrying gene therapy have successfully repaired DNA in the lungs and liver of animals with alpha-1 antitrypsin deficiency — a promising leap toward treating humans with this ...
FDA Is Investigating If Sarepta’s Gene Therapy Should Stay on Market
US Food and Drug Administration Commissioner Marty Makary said he’s “taking a hard look” at whether a gene therapy from ...
Exclusive: US FDA to request Sarepta to stop all shipments of gene therapy Elevidys, source says
The U.S. Food and Drug Administration will request Sarepta Therapeutics to voluntarily stop all shipments of its gene therapy ...
Experts call for greater physician awareness and screening of fragile X-related conditions
UC Davis MIND Institute researchers Randi and Paul Hagerman are calling for increased awareness and screening for fragile ...
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Patient dies in Sarepta gene therapy trial, adding to safety concerns
The death will likely further stoke safety concerns around Sarepta's gene therapies, as well as raise questions around the ...
Becker's Hospital Review3h
Sarepta reports 3rd patient death after gene therapy treatment
A third patient has died from acute liver failure after receiving a gene therapy treatment from Sarepta Therapeutics, the biotech company confirmed to Bloomberg on July 18. The patient died while ...
The American Journal of Managed Care3h
CGT Access Model Aims to Expand Access to Sickle Cell Treatments
Thirty-three states, the District of Columbia, and Puerto Rico will participate in CMS’ voluntary Cell and Gene Therapy (CGT) ...
BioPharma Dive3h
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 ...
Sarepta Falls After Another Patient Dies on Gene Therapy
A third patient has died this year after getting a gene therapy from Sarepta Therapeutics Inc., raising questions about the ...
Neurology Advisor3h
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 ...