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- AstraZeneca/Ionis heart-failure drug Wainua fails pivotal trial as White House reviews FDA commissioner candidatesThe White House is reviewing a shortlist of three candidates for the FDA commissioner position, according to STAT. The list includes White House adviser Heidi Overton, oncologist and health‑system…
- 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…STAT · 1mo agoRegulatoryPositive