Hematopoietic stem cell transplantation for Morquio A syndrome
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- 作者：Hiromasa Yabea ; 1 ; yabeh@is.icc.u-tokai.ac.jp" class="auth_mail" title="E-mail the corresponding author ; Akemi Tanakab ; 1 ; 2 ; akemi-chan@med.osaka-cu.ac.jp" class="auth_mail" title="E-mail the corresponding author ; Yasutsugu Chinenc ; 1 ; ychinen@med.u-ryukyu.ac.jp" class="auth_mail" title="E-mail the corresponding author ; Shunichi Katoa ; Kazuki Sawamotof ; Eriko Yasudaf ; Haruo Shintakub ; Yasuyuki Suzukid ; Tadao Oriie ; Shunji Tomatsue ; f ; stomatsu@nemours.org" class="auth_mail" title="E-mail the corresponding author
- 关键词：Morquio A syndrome ; Hematopoietic stem cell transplantation ; Respiratory function ; GVHD ; ADL
- 刊名：Molecular Genetics and Metabolism
- 出版年：2016
- 出版时间：February 2016
- 年：2016
- 卷：117
- 期：2
- 页码：84-94
- 全文大小：1807 K
文摘
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Successful four cases of HSCT are presented.
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Effect and limitation of HSCT for MPS IVA are described.
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Early HSCT provides a better therapeutic effect.
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Established bone lesion remains an unmet challenge.
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HSCT should start at an early stage prior to the irreversible bone lesion.