Caregiver experiences and observations of intrathecal idursulfase-IT treatment in a phase 2/3 trial in pediatric patients with neuronopathic mucopolysaccharidosis II

Approximately two-thirds of patients with mucopolysaccharidosis II (MPS II) have a severe, neuronopathic phenotype, characterized by somatic, cognitive, and behavioral issues. Current standard of care for the ...

Source: Orphanet Journal of Rare Diseases - March 10, 2024 Category: Internal Medicine Authors: Karen S. Yee, Sandy Lewis, Emily Evans, Carla Romano and David Alexanderian Tags: Research Source Type: research