Hematopoietic stem cell transplantation in pediatric patients with type VI mucopolysaccharidosis
HSCT aims to improve the disease and provides a permanent solution at the enzyme level, eliminating ERT. Our study showed that HSCT, a less expensive and permanent treatment option, should be offered to patients with MPS VI.
Authors
- Hayriye Daloğlu (ORCID: https://orcid.org/0009-0008-6996-0292)
- Vedat Uygun (ORCID: https://orcid.org/0000-0003-3257-7798)
- Suleimen Zhumatayev (ORCID: https://orcid.org/0000-0002-4409-8180)
- Seda Öztürkmen (ORCID: https://orcid.org/0000-0002-6713-0147)
- Koray Yalçın (ORCID: https://orcid.org/0000-0003-3454-9870)
- Gülsün Karasu (ORCID: https://orcid.org/0000-0001-5700-5919)
- Suna Çelen (ORCID: https://orcid.org/0000-0002-7930-2081)
- Akif Yeşilipek
Institutions
- Bahçeşehir University (TR)
- Antalya Eğitim ve Araştırma Hastanesi (TR)
- Istanbul Eye Hospital (TR)
- Antalya IVF (TR)
Publication Details
- Journal
- Clinical and Experimental Pediatrics
- Published
- 2025-03-11
- DOI
- https://doi.org/10.3345/cep.2024.02033
- Citations
- 2
- Primary Topic
- Lysosomal Storage Disorders Research
- Type
- article
- Field-Weighted Citation Impact
- 2.33