RGX-121 gene therapy for severe Mucopolysaccharidosis Type II (MPS II): Interim neurodevelopmental outcomes and biomarker results (4164)
Paul Harmatz, Maria L. Escolar, Can Fıçıcıoğlu, Roberto Giugliani, Yoonjin Cho, Dawn Phillips, Marie-Laure Névoret, Paulo Falabella
Abstract
To evaluate the safety and efficacy of CNS-administered gene therapy for severe MPS II
Topics & Concepts
InterimBiomarkerMedicineMucopolysaccharidosis type IIGenetic enhancementInterim analysisMucopolysaccharidosisOncologyPediatricsInternal medicineBioinformaticsGeneDiseaseGeneticsEnzyme replacement therapyBiologyClinical trialPolitical scienceLawLysosomal Storage Disorders Research