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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...
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 |
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Formato: | Online Artículo Texto |
Lenguaje: | English |
Publicado: |
Elsevier
2014
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Materias: | |
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 |
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