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- HHS launches Operation Trialblazer to accelerate U.S. drug research amid growing competition from ChinaThe Department of Health and Human Services introduced a new initiative called Operation Trialblazer aimed at speeding early-stage drug research in the United States. The plan calls for coordinate…
- Father invests $70 million in gene therapy quest for daughter with ultra‑rare NGLY1 deficiency, faces FDA scrutinyMatt Wilsey has spent a decade and roughly $70 million trying to develop a gene therapy for his daughter Grace, who suffers from NGLY1 deficiency, an ultra‑rare genetic disorder. NGLY1 deficiency…