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Gene Therapy Also Effective in Younger Children With Inherited Blood Disorders
Exa-cel is the first FDA-approved therapy utilizing CRISPR/Cas9, a type of gene editing technology through which patients' ...
Vertex Pharmaceuticals said on Saturday its gene therapy helped children aged between 5 and 11 years with sickle cell disease ...
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 ...
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 ...
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 ...
The chief medical officer of Genetix Biotherapeutics, formerly known as Bluebird bio, discusses timelines and operational ...
12don MSN
3-Year-Old Boy with Rare Condition Amazes Doctors by Becoming World's First Gene Therapy Patient
Oliver Chu was born with a rare genetic condition called Hunter syndrome that impacts physical and mental development. The ...
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