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

Fuente: Wiley Open Access

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Bibliographic Details
Main Authors:	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
Format:	Artículo Open Access
Published:	Wiley 2026
Subjects:	JIMD Reports
Online Access:	Acceder al recurso
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