Long-term open-label phase I/II extension study of intrathecal idursulfase-IT in the treatment of neuronopathic mucopolysaccharidosis II
Author:
Publisher
Elsevier BV
Subject
Genetics (clinical)
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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. Clinical characteristics and somatic burden of patients with mucopolysaccharidosis II with or without neurological involvement: An analysis from the Hunter Outcome Survey;Molecular Genetics and Metabolism Reports;2023-12
3. 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
4. Gene therapies for mucopolysaccharidoses;Journal of Inherited Metabolic Disease;2023-05-29
5. A novel mucopolysaccharidosis type II mouse model with an iduronate-2-sulfatase-P88L mutation;Scientific Reports;2023-05-15
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