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.

• Codi protocol: AZA-001-301
• Codi EudraCT: No aplica
• Grup de recerca: Neurologia Pediàtrica
• Servei: Pediatria General i Especialitats
• Investigador/a principal:  Del Toro Riera, Mireia
• Malaltia: Malalties del sistema nerviós
• Fase: Fase III