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Difficulties Associated With Enzyme Replacement Therapy for Mucopolysaccharidoses

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source: Turkish archives of pediatrics

year: 2021

authors: Yıldız Y,Sivri HS


Mucopolysaccharidoses are extremely rare, progressive, often severe multisystem disorders, some of which are managed by weekly intravenous enzyme replacement therapy. This study aimed to determine the difficulties faced by the patients with mucopolysaccharidosis and their families due to enzyme replacement therapy.

organization: Division of Pediatric Metabolism, Department of Pediatrics, Hacettepe University School of Medicine, Ankara, Turkey.

DOI: 10.5152/TurkArchPediatr.2021.21235

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