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Difficulties Associated With Enzyme Replacement Therapy for Mucopolysaccharidoses
source: Turkish archives of pediatrics
year: 2021
authors: Yıldız Y,Sivri HS
summary/abstract: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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