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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Detalles Bibliográficos
Autores principales: 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: Online Artículo Texto
Lenguaje:English
Publicado: Elsevier 2014
Materias:
The Lysosome
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4104387/
https://www.ncbi.nlm.nih.gov/pubmed/25061571
http://dx.doi.org/10.1016/j.ymgmr.2014.04.001

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4104387/
https://www.ncbi.nlm.nih.gov/pubmed/25061571
http://dx.doi.org/10.1016/j.ymgmr.2014.04.001

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