A phase I/II study of intrathecal idursulfase-IT in children with severe mucopolysaccharidosis II

2015167 citationsJournal Articlehybrid Open Access

Authors

Joseph Muenzer · University of North Carolina at Chapel Hill

Christian J. Hendriksz · Birmingham Children's Hospital

Zheng Fan · University of North Carolina at Chapel Hill

Suresh Vijayaraghavan · Birmingham Children's Hospital

V. Hugh Perry · University of North Carolina at Chapel Hill

Saikat Santra · Boston Children's Hospital

Guirish A. Solanki

Abstract

These preliminary data support further development of investigational idursulfase-IT in MPS II patients with the severe phenotype who have progressed only to a mild-to-moderate level of cognitive impairment.Genet Med 18 1, 73-81.

Topics & Keywords

Lysosomal Storage Disorders Research Spinal Dysraphism and Malformations Glycogen Storage Diseases and Myoclonus

UN Sustainable Development Goals

Good health and well-being

Publication Details

Published in: Genetics in Medicine

Volume 18, Issue 1, pp. 73-81

DOI: 10.1038/gim.2015.36

Field-Weighted Citation Impact: 12.54