FDA Advisory Committee Votes Against Zynquista for Type 1 Diabetes and Chronic Kidney Disease, Despite Support for Subgroup Benefits

FDA Advisory Committee, Zynquista (sotagliflozin), Type 1 Diabetes (T1D), Chronic Kidney Disease (CKD), Glycemic Control, SGLT1/SGLT2 Inhibitor, Lexicon Pharmaceuticals

European Commission’s Reconsideration of Translarna’s Marketing Authorization: A Critical Turning Point for Duchenne Muscular Dystrophy Treatment

Translarna, Duchenne muscular dystrophy, European Commission, EMA, CHMP, conditional marketing authorization, drug approval, regulatory decisions

FDA Advisory Committee Supports Stealth BioTherapeutics’ Elamipretide for Barth Syndrome Despite Data Concerns

Stealth BioTherapeutics, elamipretide, Barth syndrome, FDA advisory committee, ultra-rare disease, unmet medical need