MedPage Today on MSN
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 ...
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.
Two years ago, he was diagnosed with a terminal illness called Batten disease -- often leading to blindness, seizures, and ...
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 ...
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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