An 18-month double-blind, randomized, placebo-controlled, multicenter, Phase 3 study to evaluate the safety and efficacy of oral nizubaglustat (AZ-3102) in late-infantile and juvenile forms of Niemann-Pick type C disease and in late-infantile and juvenile-onset forms of GM1 gangliosidosis or GM2 gangliosidosis
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Number of participants allocated to subprotocol AZA-001-301-NPC
Timeframe: Baseline to month 18
Number of participants allocated to subprotocol AZA-001-301-GMx
Timeframe: Baseline to month 18