A phase III study to evaluate the efficacy of nizubaglustat on GM1, GM2, and NPC diseases (NAVIGATE)

Molecular Genetics and Metabolism 2026

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Authors: Laura López de Frutos, Aidan Gill, Esther Huitink, Dominique Nijkamp, Christian Freitag

DOI: 10.1016/j.ymgme.2025.109528

Cited by: 0

Fields of Study & Subjects

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