| Article ID | Journal | Published Year | Pages | File Type |
|---|---|---|---|---|
| 10833523 | Molecular Genetics and Metabolism | 2012 | 8 Pages |
Abstract
⺠HSCT showed effectiveness on brain or heart involvements in MPS II patients, when performed before a sign of brain atrophy appear in MRI for brain and before valvular regurgitation appear for heart. ⺠HSCT showed efficacy on cribriform lesion and ventricular dilatation in MRI of MPS II. ⺠Cardiac valvular regurgitation was diminished in 32% valves of HSCT-treated patients. ⺠Urinary GAG in HSCT-treated patients was lower than in ERT-treating patients.
Keywords
GAGDevelopment QuotientHSCTADLFIMMucopolysaccharidosis type IIenzyme replacement therapyMRIstandard deviationFunctional Independence Measureintelligence quotientMagnetic resonance imagingActivities of daily livingMucopolysaccharidosisSurveyMPsERTHematopoietic stem cell transplantationGlycosaminoglycan
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Authors
Akemi Tanaka, Torayuki Okuyama, Yasuyuki Suzuki, Norio Sakai, Hiromitsu Takakura, Tomo Sawada, Toju Tanaka, Takanobu Otomo, Toya Ohashi, Mika Ishige-Wada, Hiromasa Yabe, Toshihiro Ohura, Nobuhiro Suzuki, Koji Kato, Souichi Adachi, Ryoji Kobayashi,