Long-term data from a phase II study with oral nizubaglustat for late-infantile/juvenile GM2 and NPC diseases (RAINBOW)

Molecular Genetics and Metabolism 2026

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Authors: Roberto Giugliani, Daniel A.D. Valle, Dafne Dain Gandelman Horovitz, Aidan Gill, Christian Freitag, Laura López de Frutos

DOI: 10.1016/j.ymgme.2025.109432

Cited by: 0

Fields of Study & Subjects

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