Clinical and Experimental Pediatrics

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Original Article

Hematopoietic stem cell transplantation in pediatric patients with type VI mucopolysaccharidosis

Vedat Uygun, Koray Yalçın, Hayriye Daloğlu, Seda Öztürkmen, Suna Çelen, Suleimen Zhumatayev, Gülsün Karasu, Akif Yeşilipek

Background: It is uncertain whether hematopoietic stem cell transplantation (HSCT), versus standard enzyme replacement therapy (ERT), is effective for type VI mucopolysaccharidosis (MPS VI).
Purpose: New related advances in HSCT prompted an examination of the transplant procedures performed in a recent cohort. Methods: This single-center retrospective study reviewed the medical records of 17 pediatric patients with MPS VI who underwent allogeneic...

DOI: https://doi.org/10.3345/cep.2024.02033 [Accepted]

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