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...
Main Authors: | Joo-Hyun Seo, Motomichi Kosuga, Takashi Hamazaki, Haruo Shintaku, Torayuki Okuyama |
---|---|
Format: | Article |
Language: | English |
Published: |
Elsevier
2021-06-01
|
Series: | Molecular Therapy: Methods & Clinical Development |
Subjects: | |
Online Access: | http://www.sciencedirect.com/science/article/pii/S2329050121000334 |
Similar Items
-
Toileting Abilities Survey as a surrogate outcome measure for cognitive function: Findings from neuronopathic mucopolysaccharidosis II patients treated with idursulfase and intrathecal idursulfase
by: Melissa J. Hogan, et al.
Published: (2020-12-01) -
Mucopolysaccharidosis type II: Enzyme Replacement Therapy Efficiency
by: Nato D. Vashakmadze, et al.
Published: (2020-02-01) -
Natural history of cognitive development in neuronopathic mucopolysaccharidosis type II (Hunter syndrome): Contribution of genotype to cognitive developmental course
by: Joo-Hyun Seo, et al.
Published: (2020-09-01) -
EFFICACY AND SAFETY OF ENZYME REPLACEMENT THERAPY IN CHILDREN WITH MUCOPOLYSACCHARIDOSIS TYPE I, II, AND VI: A SINGLE-CENTER COHORT STUDY
by: Liliia A. Osipova, et al.
Published: (2018-04-01) -
A new strategy of desensitization in mucopolysaccharidosis type II disease treated with idursulfase therapy: A case report and review of the literature
by: Burlina, A.B, et al.
Published: (2022)