208-week outcomes of cipaglucosidase alfa plus miglustat in patients with late-onset Pompe disease treated from PROPEL baseline: Muscle function and biomarkers

Molecular Genetics and Metabolism 2026 Open access

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Authors: Tahseen Mozaffar, Drago Bratkovic, Kristl G. Claeys, Jordi Diaz-Manera, Priya S. Kishnani, Mark E. Roberts, Benedikt Schoser, Fred K. Holdbrook, Vipul Jain, Pilar Moore, Kinesha O'Brien-Prince, Antonio Toscano
DOI: 10.1016/j.ymgme.2025.109555
Cited by: 1

Fields of Study & Subjects

Lysosomal Storage Disorders Research Glycogen Storage Diseases and Myoclonus Whipple's Disease and Interleukins

Publication details

Journal / source
Molecular Genetics and Metabolism
Publisher
Elsevier BV
Volume
147
Issue
2
Pages
109555–109555
Publication date
1 February 2026
Language
EN