A phase I, multi-center, open-label study design to evaluate the safety, tolerability, pharmacokinetics, and pharmacodynamics of DNL952 in adult participants with late-onset Pompe disease

Molecular Genetics and Metabolism 2026

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Authors: Amy C. Berger, Isaac V. Cohen, Marisa S. Goo, Sarah Huntwork, Shyeilla V. Dhuria, Matthew D. Troyer, Carole Ho, Danna L. Jennings

DOI: 10.1016/j.ymgme.2025.109332

Cited by: 0

Fields of Study & Subjects

Lysosomal Storage Disorders Research Glycogen Storage Diseases and Myoclonus Pharmaceutical studies and practices