A multinational, randomized, double-blind, placebo-controlled study to assess the efficacy, pharmacodynamics, pharmacokinetics, and safety of venglustat in late onset GM2 A multinational, randomized, double-blind, placebo-controlled study to assess the efficacy, pharmaco ...
A multicenter, multinational, randomized, double-blind, placebo-controlled study to assess the efficacy, pharmacodynamics, pharmacokinetics, safety, and tolerability of venglustat in late onset GM2 gangliosidosis (Tay-Sachs disease and Sandhoff disease) together with a separate basket for juvenile/adolescent late onset GM2 gangliosidosis and ultra rare diseases within the same and similar glucosylceramide-based sphingolipid pathway A multicenter, multinational, randomized, double-blind, placebo-controlled study to assess the effic ...
Tay-Sachs Disease, Sandhoff Disease
Drug: venglustat GZ402671 - Pharmaceutical form: tablet - Route of administration: oral Drug: placebo - Pharmaceutical form: tablet - Route of administration: oral Drug: venglustatGZ402671 - Pharmaceutical form: tablet - Route of administration: oral Drug: placebo - P ...
A Multinational, Randomized, Double-blind, Placebo-controlled Study to Assess the Efficacy, Pharmacodynamics, Pharmacokinetics, and Safety of Venglustat in Late-onset GM2 A Multinational, Randomized, Double-blind, Placebo-controlled Study to Assess the Efficacy, Pharmaco ...
A Multicenter, Multinational, Randomized, Double-blind, Placebo-controlled Study to Assess the Efficacy, Pharmacodynamics, Pharmacokinetics, Safety, and Tolerability of Venglustat in Late-onset GM2 Gangliosidosis (Tay-Sachs Disease and Sandhoff Disease) Together With a Separate Basket for Juvenile/Adolescent Late-onset GM2 Gangliosidosis and Ultra-rare Diseases Within the Same and Similar Glucosylceramide-based Sphingolipid Pathway A Multicenter, Multinational, Randomized, Double-blind, Placebo-controlled Study to Assess the Effic ...
United States;Argentina;Austria;Brazil;Czechia;France;Germany;Italy;Japan;Portugal;Russian Federation;Spain;Turkey;United Kingdom United States;Argentina;Austria;Brazil;Czechia;France;Germany;Italy;Japan;Portugal;Russian Federatio ...