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TrialOrphanet journal of rare diseases2026

Sustained clinical benefit of idursulfase beta in mucopolysaccharidosis II: two-year experience from a phase 3 extension study including patients switched from idursulfase.

Minji Im et al.PubMed ↗Full text ↗Publisher ↗

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The trial

Full record →Abstract, authors, funding and every citing paper · PMID 42421116