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18-month double-blind, randomized, placebo-controlled, multicenter, Phase 3 study to evaluate the safety and efficacy of oral ni

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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  • Protocol code: AZA-001-301
  • EudraCT code: No aplica
  • Pathology: Malalties del sistema nerviós
  • Principal investigator:  Del Toro Riera, Mireia
  • Research group:  Neurologia pediàtrica
  • Service: Pediatria General i Especialitats
  • Phase: Fase III
  • Recruiting: Open
  • Status: Iniciat

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