Pharmaceutical Technology on MSN
Potentially “groundbreaking” FDA gene therapy pathway leaves key issues unresolved
A new FDA pathway could speed bespoke gene therapies, but key questions over scope and commercial viability remain.
Dr. Allyse’s research lies at the nexus of women and children’s health, health disparities, and community engagement. She has authored over a hundred academic journal articles examining bioethical ...
Born in Morristown, N.J., with virtually no immune system, Cora was diagnosed with severe combined immunodeficiency, a rare ...
The U.S. Food and Drug Administration issued on Friday a Complete Response Letter for Ultragenyx Pharmaceutical Inc.’s (NASDAQ:RARE) Biologics License Application (BLA) for UX111 (ABO-102) AAV gene ...
Andrew Joseph covers health, medicine, and the biopharma industry in Europe. You can reach Andrew on Signal at drewqjoseph.45. Finally, it seemed, Sarah Jenssen would have a chance to get a treatment ...
July 11 (Reuters) - The U.S. Food and Drug Administration has declined to approve Ultragenyx Pharmaceutical's (RARE.O), opens new tab experimental gene therapy to treat a rare inherited disorder, ...
In 1962, a pediatric neurology resident at Columbia's Neurological Institute and his colleagues in the College of Physicians and Surgeons published a detailed paper in Pediatrics describing a new ...
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