Clinical investigator perspectives on patient outcomes in children with neuronopathic mucopolysaccharidosis II during intrathecal idursulfase-IT treatment

Mucopolysaccharidosis II (MPS II) is a rare lysosomal storage disease characterized by iduronate-2-sulfatase gene (IDS) deficiency and downstream glycosaminoglycan accumulation. Two-thirds of patients present wit...

Source: Orphanet Journal of Rare Diseases - April 12, 2024 Category: Internal Medicine Authors: Karen S. Yee, David Alexanderian, Susan Martin, Bimpe Olayinka-Amao and David A. H. Whiteman Tags: Research Source Type: research