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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Main Authors:	Chinen, Yasutsugu, Higa, Takeshi, Tomatsu, Shunji, Suzuki, Yasuyuki, Orii, Tadao, Hyakuna, Nobuyuki
Format:	Online
Language:	English
Published:	Elsevier 2014
Online Access:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4292891/

id	pubmed-4292891
recordtype	oai_dc
spelling	pubmed-42928912015-01-13 Long-term therapeutic efficacy of allogenic bone marrow transplantation in a patient with mucopolysaccharidosis IVA☆ Chinen, Yasutsugu Higa, Takeshi Tomatsu, Shunji Suzuki, Yasuyuki Orii, Tadao Hyakuna, Nobuyuki Research Paper 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 has a direct impact on cartilage and bone development, resulting in systemic skeletal dysplasia. There is no curative therapy for this skeletal dysplasia. Elsevier 2014-01-14 /pmc/articles/PMC4292891/ /pubmed/25593792 http://dx.doi.org/10.1016/j.ymgmr.2013.11.002 Text en © 2013 The Authors http://creativecommons.org/licenses/by-nc-nd/3.0/ This is an open access article under the CC BY-NC-ND license (http://creativecommons.org/licenses/by-nc-nd/3.0/).
repository_type	Open Access Journal
institution_category	Foreign Institution
institution	US National Center for Biotechnology Information
building	NCBI PubMed
collection	Online Access
language	English
format	Online
author	Chinen, Yasutsugu Higa, Takeshi Tomatsu, Shunji Suzuki, Yasuyuki Orii, Tadao Hyakuna, Nobuyuki
spellingShingle	Chinen, Yasutsugu Higa, Takeshi Tomatsu, Shunji Suzuki, Yasuyuki Orii, Tadao Hyakuna, Nobuyuki Long-term therapeutic efficacy of allogenic bone marrow transplantation in a patient with mucopolysaccharidosis IVA☆
author_facet	Chinen, Yasutsugu Higa, Takeshi Tomatsu, Shunji Suzuki, Yasuyuki Orii, Tadao Hyakuna, Nobuyuki
author_sort	Chinen, Yasutsugu
title	Long-term therapeutic efficacy of allogenic bone marrow transplantation in a patient with mucopolysaccharidosis IVA☆
title_short	Long-term therapeutic efficacy of allogenic bone marrow transplantation in a patient with mucopolysaccharidosis IVA☆
title_full	Long-term therapeutic efficacy of allogenic bone marrow transplantation in a patient with mucopolysaccharidosis IVA☆
title_fullStr	Long-term therapeutic efficacy of allogenic bone marrow transplantation in a patient with mucopolysaccharidosis IVA☆
title_full_unstemmed	Long-term therapeutic efficacy of allogenic bone marrow transplantation in a patient with mucopolysaccharidosis IVA☆
title_sort	long-term therapeutic efficacy of allogenic bone marrow transplantation in a patient with mucopolysaccharidosis iva☆
description	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 has a direct impact on cartilage and bone development, resulting in systemic skeletal dysplasia. There is no curative therapy for this skeletal dysplasia.
publisher	Elsevier
publishDate	2014
url	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4292891/
_version_	1613176277656666112

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

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