Preliminary results from two trials of the gene therapy exagamglogene autotemcel (exa-cel) suggest the therapy offers an ...
The chief medical officer of Genetix Biotherapeutics, formerly known as Bluebird bio, discusses timelines and operational ...
Vertex Pharmaceuticals said on Saturday its gene therapy helped children aged between 5 and 11 years with sickle cell disease ...
13don MSN
California boy, 3, receives first-ever gene therapy for his rare disease: ‘It’s just so exciting’
A California toddler is the first person in the world to receive gene therapy to treat his devastating disease.
A type of gene therapy called CAR T that has extended survival for thousands of patients with leukemia and other blood cancers is being adapted at UC San Francisco to treat people with glioblastoma, ...
Clinical Trials Arena on MSN
Potentially “groundbreaking” FDA gene therapy pathway leaves key issues unresolved
A new FDA pathway could speed bespoke gene therapies, but key questions over scope and commercial viability remain.
Onasemnogene abeparvovec-brve is the first gene replacement therapy approved for SMA patients aged 2 years and older, expanding access beyond infants. Phase 3 trials demonstrated significant motor ...
U.S. Food and Drug Administration has approved Novartis' gene therapy for a type of rare muscle disorder, the drugmaker said ...
A new gene therapy can reverse the effects of heart failure and restore heart function in a large animal model. The therapy increases the amount of blood the heart can pump and dramatically improves ...
Novartis wins FDA approval for Itvisma, a one-time gene therapy offering broader SMA treatment with sustained motor function improvements.
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