Biohaven’s Taldefgrobep Alfa Shifts Focus to Obesity After Missing Primary Endpoint in Spinal Muscular Atrophy Phase 3 Trial

Biohaven, taldefgrobep alfa, spinal muscular atrophy (SMA), obesity, myostatin inhibitor, phase 3 trial, body composition, lean muscle mass, bone density.

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