Not logged in Intrathecal idursulfase-IT in children younger than 3 years with neuronopathic mucopolysaccharidosis II in a single-arm, open-label, phase 2/3 substudy and extension Muenzer, Joseph; Burton, Barbara K.; Harmatz, Paul; Gutierrez-solana, Luis Gonzalez; Ruiz-garcia, Matilde; Jones, Simon A.; Guffon, Nathalie; Inbar-feigenberg, Michal; Bratkovic, Drago; Rust, Stewart; Hale, Michael; Wu, Yuna; Yee, Karen S.; Whiteman, David A. H.; Alexanderian, David Share Save
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Neurodevelopmental status and adaptive behavior of pediatric patients with mucopolysaccharidosis II: a longitudinal observational study Muenzer, Joseph; Burton, Barbara K.; Amartino, Hernan M.; Harmatz, Paul R.; Gutierrez-Solana, Luis Gonzalez; Ruiz-Garcia, Matilde; Wu, Yuna; Merberg, David; Alexanderian, David; Jones, Simon A. Share Save
Intrathecal idursulfase-IT in patients with neuronopathic mucopolysaccharidosis II: Results from a phase 2/3 randomized study (vol 137, pg 127, 2022) Muenzer, Joseph; Burton, Barbara K.; Harmatz, Paul; Gutierrez-Solana, Luis Gonzalez; 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 Share Save
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Long-term open-label extension study of the safety and efficacy of intrathecal idursulfase-IT in patients with neuronopathic mucopolysaccharidosis II (vol 137, pg 22, 2022) Muenzer, Joseph; Burton, Barbara K.; Harmatz, Paul; Gonzalez-Solana, Luis; 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 Share Save
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Long-term open-label extension study of the safety and efficacy of intrathecal idursulfase-IT in patients with neuronopathic mucopolysaccharidosis II Muenzer, Joseph; Burton, Barbara K.; Harmatz, Paul; Gonzalez Gutierrez-Solana, Luis; 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 Share Save
Intrathecal idursulfase-IT in patients with neuronopathic mucopolysaccharidosis II: Results from a phase 2/3 randomized study Muenzer, Joseph; Burton, Barbara K.; Harmatz, Paul; Gonzalez Gutierrez-Solana, Luis; 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 Share Save
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Long-term safety and clinical outcomes of intrathecal heparan-N-sulfatase in patients with Sanfilippo syndrome type A Wijburg, Frits A.; Heap, Fiona; Rust, Stewart; de Ruijter, Jessica; Tump, Evelien; Marchal, Jan Pieter; Nestrasil, Igor; Shapiro, Elsa; Jones, Simon A.; Alexanderian, David Share Save
A multicenter open-label extension study of intrathecal heparan-N-sulfatase in patients with Sanfilippo syndrome type A Wijburg, Frits A.; Whitley, Chester B.; Muenzer, Joseph; Gasperini, Serena; del Toro, Mireia; Muschol, Nicole; Cleary, Maureen; Sevin, Caroline; Shapiro, Elsa; Alexanderian, David Share Save
Long-term safety and efficacy of intrathecal idursulfase-IT in patients with neuronopathic mucopolysaccharidosis type II: 2-year results from a phase 2/3 extension study Muenzer, Joseph; Burton, Barbara K.; Harmatz, Paul; Gonzalez Gutierrez-Solana, Luis; Ruiz-Garcia, Matilde; Jones, Simon A.; Guffon, Nathalie; Inbar-Feigenberg, Michal; Bratkovic, Drago; Li, Xiaoxi; Yee, Karen S.; Alexanderian, David Share Save
Comparison of cognitive function in siblings with neuronopathic mucopolysaccharidosis type II: Evaluation of early treatment with intravenous idursulfase and intrathecal idursulfase-IT Muenzer, Joseph; Burton, Barbara K.; Harmatz, Paul; Gonzalez Gutierrez-Solana, Luis; Ruiz-Garcia, Matilde; Jones, Simon A.; Guffon, Nathalie; Inbar-Feigenberg, Michal; Bratkovic, Drago; Li, Xiaoxi; Yee, Karen S.; Alexanderian, David Share Save
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Single-arm, open-label, phase 2/3 substudy and extension evaluating safety and efficacy of intrathecal idursulfase-IT in patients younger than 3 years old with neuronopathic mucopolysaccharidosis type Muenzer, Joseph; Burton, Barbara K.; Harmatz, Paul; Gonzalez Gutierrez-Solana, Luis; Ruiz-Garcia, Matilde; Jones, Simon A.; Guffon, Nathalie; Inbar-Feigenberg, Michal; Bratkovic, Drago; Li, Xiaoxi; Yee, Karen S.; Alexanderian, David Share Save
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Intrathecal heparan-N-sulfatase in patients with Sanfilippo syndrome type A: A phase IIb randomized trial Wijburg, Frits A.; Whitley, Chester B.; Muenzer, Joseph; Gasperini, Serena; del Toro, Mireia; Muschol, Nicole; Cleary, Maureen; Sevin, Caroline; Shapiro, Elsa; Bhargava, Parul; Kerr, Douglas; Alexanderian, David Share Save
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