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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Main Authors: Patel, Pravin, Suzuki, Yasuyuki, Tanaka, Akemi, Yabe, Hiromasa, Kato, Shunichi, Shimada, Tsutomu, Mason, Robert W., Orii, Kenji E., Fukao, Toshiyuki, Orii, Tadao, Tomatsu, Shunji
Formato: Artigo
Idioma:Inglês
Publicado: Elsevier 2014
Assuntos:
The Lysosome
Acceso en liña: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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