Treatment with UX111 gene therapy rapidly reduced heparansulfate (HS) exposure in cerebrospinalfluid (CSF) and improvedlong-term cognitive function in children withmucopolysaccharidosis type IIIA (MPS IIIA) - Lau, Heather A.; Patra, Kaushik; Wolf, Melissa; Smith, Nicholas J. C.; Couce, Maria L.; Rajan, Deepa; Truxal, Kristen; Castro Lopez, Maria Jose De; Fuller, Maria; Monteagudo-Vilavedra, Eines; Dunia Dougherty-De Miguel, Lucia; Riera, Mireia Del Toro; et al. (MOLECULAR GENETICS AND METABOLISM, 2025)