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

UniQure Wins First FDA RMAT in Huntington’s Disease After Strong Phase I/II Data

UniQure, Huntington’s disease, gene therapy, AMT-130, FDA, RMAT designation, Phase I/II clinical trials, neurodegenerative disorder, neurofilament light (NfL), disease progression, composite Unified Huntington’s Disease Rating Scale (cUHDRS).