Hematopoietic Stem Cell Transplantation for Morquio A Syndrome

Morquio A syndrome features systemic skeletal dysplasia. To date, there has been no curative therapy for this skeletal dysplasia. No systemic report on a long-term effect of hematopoietic stem cell transplantation (HSCT) for Morquio A has been described. We conducted HSCT for 4 cases with Morquio A...

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Detalhes bibliográficos
Publicado no:Mol Genet Metab
Main Authors: Yabe, Hiromasa, Tanaka, Akemi, Chinen, Yasutsugu, Kato, Shunichi, Sawamoto, Kazuki, Yasuda, Eriko, Shintaku, Haruo, Suzuki, Yasuyuki, Orii, Tadao, Tomatsu, Shunji
Formato: Artigo
Idioma:Inglês
Publicado em: 2015
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Article
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC5016080/
https://ncbi.nlm.nih.gov/pubmed/26452513
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.ymgme.2015.09.011
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