First-In-Human Study

Phase I First-in-Human Study with nizubaglustat (AZ-3102)

This Phase I study evaluated the safety and tolerability of nizubaglustat in healthy volunteers, including assessments of how the drug is processed in the body and its presence in plasma and cerebrospinal fluid. The study supported the clinical development of nizubaglustat for neurological lysosomal storage disorders, including GM1 and GM2 gangliosidoses and Niemann-Pick type C disease.

The study has been completed, and results have been published in the peer- reviewed journal Molecular Genetics and Metabolism.

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