On a video call in early September, Sarah Tabrizi first saw the data that she and other researchers studying Huntington’s ...
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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' ...
Backed by Italy-based Fondazione Telethon ETS, Waskyra, for Wiskott-Aldrich syndrome, is the first gene therapy from a ...
Doctors in the United Kingdom developed a groundbreaking new gene therapy that is giving hope to patients with aggressive ...
CAR7, has shown remarkable success in reversing an aggressive form of leukemia. This world-first treatment uses gene ...
The FDA on Tuesday approved etuvetidigene autotemcel (Waskyra) as the first cell-based gene therapy for patients 6 months and ...
The FDA has issued its stamp of approval to a new, cell-based option to treat Wiskott-Aldrich syndrome (WAS), marking the ...
The U.S. Food and Drug Administration said on Tuesday it had approved the first gene therapy for a rare and life-threatening ...
A new wave of gene therapy treatments offer a new lease on life for people with inherited diseases such as haemophilia. But ...
The first-of-its kind gene therapy uses a patient’s skin cells to create sheets to treat the open wounds caused by the rare, ...
Hemophilia gene therapies arrived a few years ago with blockbuster expectations. But now, one therapy has been pulled from ...
The rate of severe infections decreased from 2 infections to 0.2 infections per PYO after treatment with etuvetidigene autotemcel.
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