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

Abstract Data from a phase 2/3, randomized, controlled, open‐label, multicenter trial in children with neuronopathic mucopolysaccharidosis II (MPS II; Hunter syndrome) older than 3 years suggested a benefit of intrathecal idursulfase‐IT on cognitive functioning in some patients. We describe a separa...

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Principais autores:	Joseph Muenzer, Barbara K. Burton, Paul Harmatz, Luis González Gutiérrez‐Solana, Matilde Ruiz‐Garcia, Simon A. Jones, Nathalie Guffon, Michal Inbar‐Feigenberg, Drago Bratkovic, Stewart Rust, Michael Hale, Yuna Wu, Karen S. Yee, David A. H. Whiteman, David Alexanderian
Formato:	Artigo
Idioma:	Inglês
Publicado:	Wiley 2026-03-01
Series:	JIMD Reports
Assuntos:	cognitive function enzyme replacement therapy idursulfase infants intrathecal neuronopathic mucopolysaccharidosis II
Acceso en liña:	https://doi.org/10.1002/jmd2.12443
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