RAINBOW Study

Phase II RAINBOW Study with nizubaglustat (AZ-3102)

RAINBOW was a randomized, double-blind, placebo-controlled Phase II study evaluating the safety, tolerability, and pharmacological profile of oral nizubaglustat in patients with GM2 gangliosidosis or Niemann-Pick type C disease. The study aimed to determine appropriate dosing and assess how the drug is processed and acts in the body.

The study has been completed and supports the continued clinical development of nizubaglustat.

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