Treatment with UX111 Gene Therapy Rapidly Reduced Heparan Sulfate (HS) Exposure in Cerebrospinal Fluid (CSF) and Improved Long-term Cognitive Function in Children with Mucopolysaccharidosis IIIA (MPS IIIA)

Large Cohort Integrative Multiomic Analyses Identifies Novel Risk Genes for Congenital Cerebral Ventriculomegaly and Characterizes Distinct Spatiotemporal Clusters of Pathogenesis

Children with Peri-Optic Neuritis Have Favorable Visual Outcomes After Steroid Treatment: A Singe-Center Pediatric Case Series

Subgroup and Subdomain Analysis of Patients with Long Term Follow-Up In a Phase III, Randomized, Placebo-Controlled Crossover Trial with Levacetylleucine (IB1001) for Niemann-Pick Disease Type C