90-month physician and patient-reported outcomes with cipaglucosidase alfa plus miglustat (cipa + mig) in adults with Pompe disease in ATB200-02, an open-label phase I/II study

Molecular Genetics and Metabolism 2026

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Authors: Mark E. Roberts, Barry J. Byrne, Paula R. Clemens, Özlem Göker-Alpan, Priya S. Kishnani, Tahseen Mozaffar, Benedikt G. H. Schoser, Nadine van der Beek, Farah Amon, Brian Fox, Fred K. Holdbrook, Vipul Jain
+1 more Drago Bratkovic
DOI: 10.1016/j.ymgme.2025.109597
Cited by: 0

Fields of Study & Subjects

Lysosomal Storage Disorders Research Glycogen Storage Diseases and Myoclonus Biomedical Research and Pathophysiology

Publication details

Journal / source
Molecular Genetics and Metabolism
Volume
147
Issue
2
Pages
109597–109597
Publication date
1 February 2026
Language
EN