Intrathecal idursulfase-IT in patients with neuronopathic mucopolysaccharidosis II: Results from a phase 2/3 randomized study-Reference-Cited by-同舟云学术

Intrathecal idursulfase-IT in patients with neuronopathic mucopolysaccharidosis II: Results from a phase 2/3 randomized study

Published:2022-09 Issue:1-2 Volume:137 Page:127-139
ISSN:1096-7192
Container-title:Molecular Genetics and Metabolism
language:en
Short-container-title:Molecular Genetics and Metabolism

Author:

Muenzer Joseph,Burton Barbara K.,Harmatz Paul,Gutiérrez-Solana Luis González,Ruiz-Garcia Matilde,Jones Simon A.,Guffon Nathalie,Inbar-Feigenberg Michal,Bratkovic Drago,Hale Michael,Wu Yuna,Yee Karen S.,Whiteman David A.H.,Alexanderian David

Publisher

Elsevier BV

Subject

Endocrinology,Genetics,Molecular Biology,Biochemistry,Endocrinology, Diabetes and Metabolism

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1. Intracerebroventricular enzyme replacement therapy in patients with neuronopathic mucopolysaccharidosis type II: Final report of 5-year results from a Japanese open-label phase 1/2 study;Molecular Genetics and Metabolism;2023-12

2. Frequency of iduronate-2-sulfatase gene variants detected in newborn screening for mucopolysaccharidosis type II in Japan;Molecular Genetics and Metabolism Reports;2023-12

3. Neurodevelopmental status and adaptive behavior of pediatric patients with mucopolysaccharidosis II: a longitudinal observational study;Orphanet Journal of Rare Diseases;2023-11-16

4. A post hoc analysis of Projected Retained Ability Scores (PRAS) for the longitudinal assessment of cognitive functioning in patients with neuronopathic mucopolysaccharidosis II receiving intrathecal idursulfase-IT;Orphanet Journal of Rare Diseases;2023-11-02

5. Corrigendum to: Intrathecal idursulfase-IT in patients with neuronopathic mucopolysaccharidosis II: Results from a phase 2/3 randomized study;Molecular Genetics and Metabolism;2023-11