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

Internet

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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