Why the FDA extended its adrabetadex review and what it means for Beren Therapeutics
Adrabetadex faces a longer FDA review in infantile-onset NPC. The delay could shape rare-disease drug standards in 2026.
Neurocrine’s CAHtalyst pediatric data sharpens the case for CRENESSITY durability
Steroid reduction is the real test in pediatric CAH. Neurocrine’s CRENESSITY data now faces the durability, access and adoption question.
Can NovelMed Therapeutics challenge established PNH drugs with its Bb-targeting antibody Ruxoprubart?
NovelMed Therapeutics advances Ruxoprubart for paroxysmal nocturnal hemoglobinuria with Phase II data and a new subcutaneous trial. Discover what comes next.
Can Idorsia Pharmaceuticals Ltd’s lucerastat redefine Fabry disease treatment beyond enzyme replacement therapy?
Discover how Idorsia Pharmaceuticals Ltd’s FDA-aligned Phase 3 plan could make lucerastat the first oral therapy for all Fabry disease patients. Read the analysis.