Open access
Sep 2026
Longitudinal Assessment of Cognitive Development in 23 Patients With Mucopolysaccharidosis (MPS) Type II: Results of up to 14 Years of Follow‐Up
This study investigated long‐term cognitive development and genotype–phenotype relationships in patients with Mucopolysaccharidosis Type II (MPS II). A nationwide prospective cohort study was conducted in the Netherlands with cognitive follow‐up since 2007. Patients were classified as neuronopathic or non‐neuronopathic...
J. Holdorp, M. E. Michelle Kruijshaar, A. A. M. Audrey Vollebregt et al.
· Journal of Inherited Metabol... · 0 citations