Long-term therapeutic efficacy of allogenic bone marrow transplantation in a patient with mucopolysaccharidosis IVA

Long-term therapeutic efficacy of allogenic bone marrow transplantation in a patient with mucopolysaccharidosis IVA

Mucopolysaccharidosis IVA (MPS IVA) is one of the lysosomal storage diseases. It is caused by the deficiency of N-acetylgalactosamine-6-sulfate sulfatase. Deficiency of this enzyme leads to accumulation of the specific glycosaminoglycans keratan sulfate and chondroitin-6-sulfate. This accumulation h...

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Bibliographic Details
Main Authors: Yasutsugu Chinen, Takeshi Higa, Shunji Tomatsu, Yasuyuki Suzuki, Tadao Orii, Nobuyuki Hyakuna
Format: Article
Language:English
Published: Elsevier 2014-01-01
Series:Molecular Genetics and Metabolism Reports
Subjects:
Mucopolysaccharidosis IVA
Bone marrow transplantation
GALNS
Respiratory function
Bone mineral density
Online Access:http://www.sciencedirect.com/science/article/pii/S2214426913000104

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http://www.sciencedirect.com/science/article/pii/S2214426913000104

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