Dec 6 (Reuters) - Vertex Pharmaceuticals said on Saturday its gene therapy helped children aged between 5 and 11 years with ...
Investor optimism has waned as final minutes from uniQure’s pre-BLA meeting with the FDA convey that data from the company’s ...
Three years after introducing Hemgenix, the first gene therapy for hemophilia B, CSL Behring has released long-term data ...
Encoded Therapeutics has linked its Dravet syndrome gene therapy ETX101 to a 78% reduction in seizures, keeping the biotech ...
Preliminary results from two trials of the gene therapy exagamglogene autotemcel (exa-cel) suggest the therapy offers an ...
Four decades have passed since cochlear implants gave infants born deaf the ability to hear. Now, gene therapy promises to ...
Regeneron is adding its name to Tessera Therapeutics’ unique in vivo gene writing program, inking a deal that lets it in on a ...
The chief medical officer of Genetix Biotherapeutics, formerly known as Bluebird bio, discusses timelines and operational ...
Harlow is defying the odds after receiving groundbreaking gene therapy for a rare brain disorder. Her progress has been called "a miracle" by her family ...
Two years ago, he was diagnosed with a terminal illness called Batten disease -- often leading to blindness, seizures, and ...
Novartis is pricing Itvisma at a wholesale acquisition cost of $2.59 million, a company spokesperson told Fierce Pharma. At ...
A study assessing the real-world commercial roll-out of gene therapies for sickle cell disease and beta thalassemia offers ...
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