Early enzyme replacement therapy enables a successful hematopoietic stem cell transplantation in mucopolysaccharidosis type IH: Divergent clinical outcomes in two Japanese siblings

Mucopolysaccharidosis type IH (MPS IH, Hurler syndrome) is a progressive, multisystem autosomal recessive lysosomal storage disorder resulting in the consequent accumulation of glycosaminoglycans. It is well recognized that early hematopoietic stem cell transplantation (HSCT) prevents neurocognitive...

Ausführliche Beschreibung

Bibliographische Detailangaben

Veröffentlicht in:	Brain & development (Tokyo. 1979) 2019-06, Vol.41 (6), p.546-550
Hauptverfasser:	Yamazaki, Narutoshi, Kosuga, Motomichi, Kida, Kazuhiro, Takei, Go, Fukuhara, Yasuyuki, Matsumoto, Hiroshi, Senda, Masayoshi, Honda, Akihito, Ishiguro, Akira, Koike, Takashi, Yabe, Hiromasa, Okuyama, Torayuki
Format:	Artikel
Sprache:	eng
Schlagworte:	Central nervous system deterioration Enzyme replacement therapy Hematopoietic stem cell transplantation Mucopolysaccharidosis type IH
Online-Zugang:	Volltext
Tags:	Tag hinzufügen Keine Tags, Fügen Sie den ersten Tag hinzu!