Intrathecal idursulfase-IT in patients with neuronopathic mucopolysaccharidosis II: Results from a phase 2/3 randomized study

Autor: Muenzer, Joseph, Burton, Barbara K., Harmatz, Paul, Gutiérrez-Solana, Luis González, 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
Zdroj: In Molecular Genetics and Metabolism September-October 2022 137(1-2):127-139
Databáze: ScienceDirect
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