An age subgroup analysis of the phase 1/2 study of intravenous tividenofusp alfa for mucopolysaccharidosis type II

Molecular Genetics and Metabolism 2026

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Authors: Paul Harmatz, Joseph Muenzer, Barbara K. Burton, Deepa Rajan, Simon A. Jones, Johanna M. P. van den Hout, John J. Mitchell, Matthew D. Troyer, Natalie J. Engmann, Fabian Model, Akhil Bhalla, Imanol Zubizarreta
+3 more Mostafa Wali, Peter S. Chin, Carole Ho
DOI: 10.1016/j.ymgme.2026.110002
Cited by: 1

Fields of Study & Subjects

Lysosomal Storage Disorders Research Glycogen Storage Diseases and Myoclonus Amyloidosis: Diagnosis Treatment Outcomes

Publication details

Journal / source
Molecular Genetics and Metabolism
Publisher
Elsevier BV
Volume
148
Issue
1
Pages
110002–110002
Publication date
1 May 2026
Language
EN