Long-term somatic efficacy of pabinafusp alfa across a broad spectrum of age groups and phenotypes in patients with mucopolysaccharidosis type II

Molecular Genetics and Metabolism 2026

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Authors: Ana Margarida Martins, Roberto Giugliani, Kimitoshi Nakamura, Norio Sakai, Motomichi Kosuga, Toshiaki Ikeda, Robin D. LeWinter, John A. Sharpe, Hideaki Hirai, Ricardo Cunegundes, Florin Floricel, Takashi Hamazaki
DOI: 10.1016/j.ymgme.2025.109543
Cited by: 0

Fields of Study & Subjects

Lysosomal Storage Disorders Research Hemophilia Treatment and Research Neurogenetic and Muscular Disorders Research

Publication details

Journal / source
Molecular Genetics and Metabolism
Volume
147
Issue
2
Pages
109543–109543
Publication date
1 February 2026
Language
EN