Development of idursulfase therapy for mucopolysaccharidosis type II (Hunter syndrome): the past, the present and the future
Author:
Publisher
Informa UK Limited
Subject
Drug Discovery,Pharmaceutical Science,Pharmacology
Cited by 71 articles. 订阅此论文施引文献 订阅此论文施引文献，注册后可以免费订阅5篇论文的施引文献，订阅后可以查看论文全部施引文献
1. Evaluation of early treatment with intravenous idursulfase and intrathecal idursulfase‐IT on cognitive function in siblings with neuronopathic mucopolysaccharidosis II;Journal of Inherited Metabolic Disease;2024-09-09
2. Intravenous Idursulfase for the Treatment of Mucopolysaccharidosis Type II: A Systematic Literature Review;International Journal of Molecular Sciences;2024-08-06
3. Clinical investigator perspectives on patient outcomes in children with neuronopathic mucopolysaccharidosis II during intrathecal idursulfase-IT treatment;Orphanet Journal of Rare Diseases;2024-04-12
4. Advancing clinical development for neuronopathic Hunter syndrome through a quantitatively‐driven reverse translation framework;Clinical and Translational Science;2024-03-28
5. Comparative dose effectiveness of intravenous and intrathecal AAV9.CB7.hIDS, RGX-121, in mucopolysaccharidosis type II mice;Molecular Therapy - Methods & Clinical Development;2024-03
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