Update on FORTIS: A phase 1/2 open-label clinical trial on AT845 gene replacement therapy for late-onset Pompe disease

Molecular Genetics and Metabolism 2026

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Authors: Jordi Díaz‐Manera, Nicola Longo, Mark Walzer, Gurunadh Reddy Chichili, Sean Yiu, David H. Viskochil, Chieri Hayashi, Tahseen Mozaffar

DOI: 10.1016/j.ymgme.2025.109386

Cited by: 0

Fields of Study & Subjects

Lysosomal Storage Disorders Research Glycogen Storage Diseases and Myoclonus Virus-based gene therapy research