TURKISH ARCHIVES OF PEDIATRICS, cilt.56, sa.6, ss.602-609, 2021 (ESCI)
Background: Mucopolysaccharidoses are extremely rare, progressive, often severe multi-system disorders, some of which are managed by weekly intravenous enzyme replacement therapy. This study aimed to determine the difficulties faced by the patients with mucopolysac-charidosis and their families due to enzyme replacement therapy.