Researchers at the University of Geneva, together with colleagues in Switzerland, France, the United States and Israel, ...
A cutting-edge therapy using base-edited immune cells is offering a major breakthrough for patients with one of the toughest ...
Sarepta Therapeutics (NASDAQ:SRPT) leads in genetic therapies, contributing to ongoing discussions surrounding the nasdaq ...
Rocket Pharmaceuticals (NASDAQ:RCKT) advances gene-therapy innovation and appears in broader discussions involving the nasdaq ...
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 ...
Nov 24 (Reuters) - (This Nov 24 story has been corrected to fix the pricing for Zolgensma to $2.5 million from $2.1 million in paragraph 4) The U.S. Food and Drug Administration has approved Novartis' ...
Copyright 2025 The Associated Press. All Rights Reserved. Copyright 2025 The Associated Press. All Rights Reserved. The name and logo of Sarepta Therapeutics is ...
The FDA has officially limited the label of Sarepta Therapeutics’ Elevidys, putting an end to a whirlwind few months that saw the abrupt departure—and reinstatement—of top agency official Vinay Prasad ...
Through substantial leadership turnover and workforce cuts, the FDA has continued to support the advanced therapy sector, actively working to remove obstacles to innovation. The cell and gene therapy ...
In a highly anticipated article, FDA officials Vinay Prasad, M.D., and Martin Makary, M.D., outlined a novel regulatory pathway that could trigger a seismic shift in how bespoke gene editing therapies ...
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