Difficulties Associated with Enzyme Replacement Therapy for Mucopolysaccharidoses
TURKISH ARCHIVES OF PEDIATRICS, cilt.56, sa.6, ss.602-609, 2021 (ESCI, TRDizin)
- 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.