Scientists have successfully transplanted gene-edited insulin-producing cells into a man with type 1 diabetes—allowing him to ...
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 ...
Huntington’s disease is one of the cruelest genetic afflictions a family can endure. The condition, which affects around 41,000 Americans, is the result of an error ...
A three-year-old Oliver (Ollie) Chu has become the world’s first patient to receive a revolutionary stem cell gene therapy for Hunter syndrome. This rare, progressive, inherited disorder damages the ...
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' ...
Mixed headlines have plagued the cell and gene therapy space of late. We believe that a renewed case of optimism is not only warranted but essential if these therapies are to reach their full ...
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 ...
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