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Impact of enzyme replacement therapy and hematopoietic stem cell therapy on growth in patients with Hunter syndrome

Patients with Hunter syndrome (mucopolysaccharidosis II) present with skeletal dysplasia including short stature as well as CNS and visceral organ involvement. A previous study on Hunter syndrome indicated an impact on brain and heart involvement after hematopoietic stem cell therapy (HSCT) at an ea...

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Κύριοι συγγραφείς:	Patel, Pravin, Suzuki, Yasuyuki, Tanaka, Akemi, Yabe, Hiromasa, Kato, Shunichi, Shimada, Tsutomu, Mason, Robert W., Orii, Kenji E., Fukao, Toshiyuki, Orii, Tadao, Tomatsu, Shunji
Μορφή:	Artigo
Γλώσσα:	Inglês
Έκδοση:	Elsevier 2014
Θέματα:	The Lysosome
Διαθέσιμο Online:	https://ncbi.nlm.nih.gov/pmc/articles/PMC4104387/ https://ncbi.nlm.nih.gov/pubmed/25061571 https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.ymgmr.2014.04.001
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Impact of enzyme replacement therapy and hematopoietic stem cell therapy on growth in patients with Hunter syndrome

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