On a video call in early September, Sarah Tabrizi first saw the data that she and other researchers studying Huntington’s ...
JABSOM Cell and Molecular Biology researcher Dr. Jesse Owens has spent the better part of two decades chasing a vision that ...
DMD gene therapy faces challenges in safety, efficacy, and long-term outcomes, despite FDA approval of delandistrogene moxeparvovec. Future strategies focus on novel AAV capsids, dual/triple vector ...
ZURICH (Reuters) - Roche is buying U.S.-based gene therapy specialist Spark Therapeutics for $4.3 billion after developments in this area convinced the Swiss drugmaker to "step up", Chief Executive ...
Alison Clare has received funding from the Wellcome Trust and the Medical Research Council. Her position is currently funded by National Institute for Health and Care Research (NIHR) Biomedical ...
Vence L. Bonham Jr., Acting Deputy Director at the National Human Genome Research Institute (NHGRI) and an investigator in the National Human Genome Research Institute’s (NHGRI) Division of Intramural ...
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'Will it really work?': Young sickle cell patient among the first to start new gene therapy
WASHINGTON — For Wedam Minyila, hospital rooms have always meant blinding pain. “Like someone is jamming a knife in me,” he said. But for a brief moment on a recent December morning, Wedam, 19, who ...
A study assessing the real-world commercial roll-out of gene therapies for sickle cell disease and beta thalassemia offers ...
ROME, Italy, Dec. 10, 2025 /PRNewswire/ -- Fondazione Telethon announced today that the U.S. Food and Drug Administration (FDA) has approved the Biologics License Application (BLA) for Waskyra, an ex ...
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