The U.S. Food and Drug Administration said on Tuesday it had approved the first gene therapy for a rare and life-threatening ...
The FDA has granted approval to a new gene therapy for a rare immune disorder, the maker of which is notably not a drug ...
Revolutions in gene therapy are rapidly changing the landscape of modern medicine, forcing society and science alike to ...
FDA approves 1st gene therapy for Wiskott-Aldrich syndrome, offering a new treatment for patients lacking matched stem cell donors.
Zevaskyn, the first gene therapy for recessive dystrophic epidermolysis bullosa, showed significant wound healing in phase 3 ...
The FDA has issued its stamp of approval to a new, cell-based option to treat Wiskott-Aldrich syndrome (WAS), marking the ...
A cutting-edge base-edited CAR T-cell therapy is opening a revolutionary new frontier for patients battling aggressive T-cell ...
NEW YORK and TRUMBULL, Conn., April 30, 2025 /PRNewswire/ -- Apertura Gene Therapy, a biotechnology company focused on innovative gene therapy solutions, and the Rett Syndrome Research Trust (RSRT), ...
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, ...
Kids and teenagers with spinal muscular atrophy had more mobility after receiving a single-dose gene replacement therapy, ...
Novartis’ Itvisma wins FDA approval as the first gene replacement therapy for spinal muscular atrophy (SMA) patients age 2 ...
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 ...
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