BioMarin’s Brineura Receives Significant Label Expansion from FDA for Children Under 3 with CLN2 Disease

BioMarin, Brineura, FDA, Label Expansion, CLN2 Disease, Children Under 3, Cerliponase Alfa, Intraventricular Injection, Motor Ability Deterioration, Neurodegenerative Symptoms

AstraZeneca’s Ultomiris Gains FDA Approval for Rare Autoimmune Condition – NMOSD

AstraZeneca, Ultomiris, ravulizumab-cwvz, FDA Approval, Rare Autoimmune Disease, Neuromyelitis Optica Spectrum Disorder (NMOSD), Long-acting C5 complement inhibitor, Phase 3 CHAMPION-NMOSD Trial, Reduced Relapse Rate, Dosing Frequency