ROSSELLA: An ongoing open-label, multicenter, global trial to study next-generation treatment of infantile-onset Pompe disease combining enzyme replacement with a stabilizing iminosugar

Molecular Genetics and Metabolism 2026

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Authors: Thorsten Marquardt, Sheela Sitaraman Das, Kinesha O'Brien-Prince, Stuart Black

DOI: 10.1016/j.ymgme.2025.109542

Cited by: 0

Fields of Study & Subjects

Lysosomal Storage Disorders Research Glycogen Storage Diseases and Myoclonus Metabolism and Genetic Disorders