Long-Term Follow-up Posthematopoietic Stem Cell Transplantation in a Japanese Patient with Type-VII Mucopolysaccharidosis

• Published in: Diagnostics (Basel) Vol. 10; no. 2; p. 105
• Main Authors: Orii, Kenji, Suzuki, Yasuyuki, Tomatsu, Shunji, Orii, Tadao, Fukao, Toshiyuki
• Format: Journal Article
• Language: English
• Published: Switzerland MDPI 16.02.2020; MDPI AG
• Subjects: activity of daily living; Case Report; hematopoietic stem cell transplantation; mucopolysaccharidoses; sly syndrome; hematopoietic stem cell transplantation; activity of daily living; mucopolysaccharidoses; Sly syndrome

The effectiveness of hematopoietic stem cell transplantation (HSCT) for type-VII mucopolysaccharidosis (MPS VII, Sly syndrome) remains controversial, although recent studies have shown that it has a clinical impact. In 1998, Yamada et al. reported the first patient with MPS VII, who underwent HSCT a...