The U.S. Food and Drug Administration has issued a supplemental approval for Casgevy (exagamglogene autotemcel) for patients ...
The development of a pioneering gene therapy to treat a devastating, rare immune disorder led by UCL scientists is set to ...
A young man from southeastern Louisiana has become the first in his region to be functionally cured of sickle cell disease, according to his medical team, paving the way for him to pursue his dream of ...
Morning Overview on MSN
The FDA just approved the first-ever gene therapy for inherited hearing loss
Children and adults born with severe hearing loss caused by mutations in the OTOF gene now have a treatment option that did ...
Onasemnogene abeparvovec gene therapy post-nusinersen or risdiplam showed meaningful motor improvements in children with SMA, with a manageable safety profile. The study cohort included older, heavier ...
Recent data published in NEJM demonstrates that CRISPR gene-editing therapy achieves complete clinical efficacy endpoints in ...
A child enrolled in Capsida Biotherapeutics’ gene therapy trial for STXBP1 developmental and epileptic encephalopathy has died, prompting the company to pause the study while investigating the death.
A gene therapy that instructs cells to produce more of an anti-ageing protein called klotho is about to be offered by a US company at overseas clinics to bypass FDA rules ...
Four decades have passed since cochlear implants gave infants born deaf the ability to hear. Now, gene therapy promises to restore natural hearing for those born with a rare form of deafness, and the ...
Forbes contributors publish independent expert analyses and insights. A patient in a late-stage gene editing trial for a rare heart condition died from fatal liver complications after receiving an ...
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