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National clinical trial(s)
AUSTRIA
WIEN
ADDRESS: NOT PROVIDED - AT
AMETHIST: 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) - AT
Institution: Information not provided - AT
GERMANY
Berlin
ADDRESS: NOT PROVIDED - DE
AMETHIST: 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) - DE
Institution: Information not provided - DE
ITALY
EMILIA ROMAGNA
ADDRESS: NOT PROVIDED - IT
AMETHIST: 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) - IT
Institution: Information not provided - IT
SPAIN
Cataluña
L'HOSPITALET DE LLOBREGAT
Effects of N-Acetyl-L-Leucine on GM2 Gangliosidosis (Tay-Sachs and Sandhoff Disease): A multinational, multicenter, open-label, rater-blinded Phase II study -ES
Hospital Universitari de Bellvitge
Servicio de Neurología
SPAIN
Madrid
ADDRESS: NOT PROVIDED - ES
AMETHIST: 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) [...] glucosylceramide-based sphingolipid pathway -ES
Institution: Information not provided - ES
Multinational clinical trial(s)
UNITED KINGDOM
Greater Manchester
ADDRESS: NOT PROVIDED - UK
SAVVY CHILD: Phase I/II open-label trial to determine the safety and tolerability of intracranial gene therapy in GM2 gangliosidosis using recombinant adeno-associated viral vectors
Institution: Information not provided - UK
UNITED KINGDOM
Oxfordshire
BEGBROKE
UNITED STATES
Massachusetts
CAMBRIDGE