Difficulties Associated with Enzyme Replacement Therapy for Mucopolysaccharidoses

YILDIZ Y., SİVRİ H. S.

TURKISH ARCHIVES OF PEDIATRICS, cilt.56, sa.6, ss.602-609, 2021 (ESCI) identifier identifier identifier

  • Yayın Türü: Makale / Tam Makale
  • Cilt numarası: 56 Sayı: 6
  • Basım Tarihi: 2021
  • Doi Numarası: 10.5152/turkarchpediatr.2021.21235
  • Dergi Adı: TURKISH ARCHIVES OF PEDIATRICS
  • Derginin Tarandığı İndeksler: Emerging Sources Citation Index (ESCI), TR DİZİN (ULAKBİM)
  • Sayfa Sayıları: ss.602-609
  • Anahtar Kelimeler: 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 Üniversitesi Adresli: Evet

Özet

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.