P169: 90-month efficacy outcomes with cipaglucosidase alfa plus miglustat in adults with Pompe disease in ATB200-02: An open-label phase I/II study

Genetics in Medicine Open 2026 Open access

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Authors: Priya Kishnani, Drago Bratkovic, Barry J. Byrne, Paula R. Clemens, Özlem Göker-Alpan, Tahseen Mozaffar, Mark Roberts, Nadine van der Beek, Farah Amon, Brian Fox, Fred K. Holdbrook, Vipul Jain
+1 more Benedikt G. H. Schoser
DOI: 10.1016/j.gimo.2026.103663
Cited by: 0

Fields of Study & Subjects

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

Publication details

Journal / source
Genetics in Medicine Open
Volume
4
Pages
103663–103663
Publication date
1 January 2026
Language
EN