Backed by Italy-based Fondazione Telethon ETS, Waskyra, for Wiskott-Aldrich syndrome, is the first gene therapy from a ...
An experimental gene therapy treatment created at UCLA gave a "bubble girl" born with a rare genetic disorder a new life.
The FDA has issued its stamp of approval to a new, cell-based option to treat Wiskott-Aldrich syndrome (WAS), marking the ...
A single-dose gene replacement therapy is found to improve movement ability in children over 2 years of age and teenagers with spinal muscular atrophy, according to research published in Nature ...
On a video call in early September, Sarah Tabrizi first saw the data that she and other researchers studying Huntington’s ...
A cutting-edge base-edited CAR T-cell therapy is opening a revolutionary new frontier for patients battling aggressive T-cell ...
Rocket Pharmaceuticals (NASDAQ:RCKT) advances gene-therapy innovation and appears in broader discussions involving the nasdaq ...
Sarepta Therapeutics (NASDAQ:SRPT) leads in genetic therapies, contributing to ongoing discussions surrounding the nasdaq ...
Researchers at the University of Geneva, together with colleagues in Switzerland, France, the United States and Israel, ...
Scientists have successfully transplanted gene-edited insulin-producing cells into a man with type 1 diabetes—allowing him to ...
According to the Pan American Health Organization, 7 out of 10 deaths in people over 70 are from non-communicable diseases (NCDs). Aging itself is the single greatest risk factor for the majority of ...
Study has identified genetic variants in lung-related proteins may affect eye development in preterm infants, altering risk of retinopathy of prematurity.
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