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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Bibliografiske detaljer
Udgivet i:Mol Genet Metab Rep
Main Authors: Chinen, Yasutsugu, Higa, Takeshi, Tomatsu, Shunji, Suzuki, Yasuyuki, Orii, Tadao, Hyakuna, Nobuyuki
Format: Artigo
Sprog:Inglês
Udgivet: Elsevier 2014
Fag:
Research Paper
Online adgang:https://ncbi.nlm.nih.gov/pmc/articles/PMC4292891/
https://ncbi.nlm.nih.gov/pubmed/25593792
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.ymgmr.2013.11.002
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