Long-term efficacy of hematopoietic stem cell transplantation on brain involvement in patients with mucopolysaccharidosis type II: A nationwide survey in Japan

Hematopoietic stem cell transplantation (HSCT) has not been indicated for patients with mucopolysaccharidosis II (MPS II, Hunter syndrome), while it is indicated for mucopolysaccharidosis I (MPS I) patients

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Veröffentlicht in:	Molecular genetics and metabolism 2012-11, Vol.107 (3), p.513-520
Hauptverfasser:	Tanaka, Akemi, Okuyama, Torayuki, Suzuki, Yasuyuki, Sakai, Norio, Takakura, Hiromitsu, Sawada, Tomo, Tanaka, Toju, Otomo, Takanobu, Ohashi, Toya, Ishige-Wada, Mika, Yabe, Hiromasa, Ohura, Toshihiro, Suzuki, Nobuhiro, Kato, Koji, Adachi, Souichi, Kobayashi, Ryoji, Mugishima, Hideo, Kato, Shunichi
Format:	Artikel
Sprache:	eng
Schlagworte:	Activities of Daily Living Brain - drug effects Brain - enzymology Brain - pathology Brain efficacy Child Child, Preschool Enzyme Replacement Therapy Female Glycosaminoglycans - urine Health Care Surveys Hematopoietic Stem Cell Transplantation Humans Iduronidase - therapeutic use Japan Magnetic Resonance Imaging Male Mitral Valve Insufficiency - enzymology Mitral Valve Insufficiency - pathology Mitral Valve Insufficiency - prevention & control Mucopolysaccharidosis II - enzymology Mucopolysaccharidosis II - pathology Mucopolysaccharidosis II - therapy Mucopolysaccharidosis type II Retrospective Studies Secondary Prevention Survey Time Treatment Outcome Young Adult
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Beschreibung
Zusammenfassung:	Hematopoietic stem cell transplantation (HSCT) has not been indicated for patients with mucopolysaccharidosis II (MPS II, Hunter syndrome), while it is indicated for mucopolysaccharidosis I (MPS I) patients
ISSN:	1096-7192 1096-7206
DOI:	10.1016/j.ymgme.2012.09.004