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Impact of intracerebroventricular enzyme replacement therapy in patients with neuronopathic mucopolysaccharidosis type II

This open-label, phase 1/2 study (JMACCT CTR JMA-IIA00350) evaluated the efficacy and safety of intracerebroventricular idursulfase beta in patients with mucopolysaccharidosis II (MPS II). Herein, we report the 100-week results. Six patients with severe MPS II aged 23–65 months were enrolled. Idursu...

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Detalles Bibliográficos
Autores principales:	Seo, Joo-Hyun, Kosuga, Motomichi, Hamazaki, Takashi, Shintaku, Haruo, Okuyama, Torayuki
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	American Society of Gene & Cell Therapy 2021
Materias:	Original Article
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7957024/ https://www.ncbi.nlm.nih.gov/pubmed/33768130 http://dx.doi.org/10.1016/j.omtm.2021.02.018

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