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- Regeneron's Pasatru receives FDA approval for ultra-rare bone-growth disease FOPRegeneron Pharmaceuticals has secured FDA approval for Pasatru, a therapy aimed at treating fibrodysplasia ossificans progressiva (FOP), an ultra‑rare condition in which bone forms in soft tissues.…
- FDA grants accelerated approval to Genglycos, first therapy for glycogen storage disease type IaThe U.S. Food and Drug Administration issued an accelerated approval for Genglycos (pariglasgene brecaparvovec-opnr), marking the first therapy for patients aged eight years and older with glycogen…