Copy For Citation
YILDIZ Y., SİVRİ H. S.
TURKISH ARCHIVES OF PEDIATRICS, vol.56, no.6, pp.602-609, 2021 (ESCI)
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Publication Type:
Article / Article
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Volume:
56
Issue:
6
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Publication Date:
2021
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Doi Number:
10.5152/turkarchpediatr.2021.21235
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Journal Name:
TURKISH ARCHIVES OF PEDIATRICS
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Journal Indexes:
Emerging Sources Citation Index (ESCI), TR DİZİN (ULAKBİM)
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Page Numbers:
pp.602-609
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Keywords:
Burden of disease, caregiver burden, enzyme replacement therapy, inborn errors of metabolism, lysosomal storage diseases, mucopolysaccharidosis, questionnaire, Turkey, HOME INFUSION, CHALLENGES, VI, DISORDERS, SAFE
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Hacettepe University Affiliated:
Yes
Abstract
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.