Difficulties Associated with Enzyme Replacement Therapy for Mucopolysaccharidoses

YILDIZ Y., SİVRİ H. S.

TURKISH ARCHIVES OF PEDIATRICS, vol.56, no.6, pp.602-609, 2021 (ESCI) identifier identifier identifier

  • Publication Type: Article / Article
  • Volume: 56 Issue: 6
  • Publication Date: 2021
  • Doi Number: 10.5152/turkarchpediatr.2021.21235
  • Journal Name: TURKISH ARCHIVES OF PEDIATRICS
  • Journal Indexes: Emerging Sources Citation Index (ESCI), TR DİZİN (ULAKBİM)
  • Page Numbers: pp.602-609
  • 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
  • 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.