Clinical Trials on Gangliosidosis, GM1 in Spain

Total 4 results

Idorsia Pharmaceuticals Ltd.

Completed

Natural History Study for Pediatric Patients With Early Onset of Either GM1 Gangliosidosis, GM2 Gangliosidoses, or Gaucher Disease Type 2 (RETRIEVE)

GM2 Gangliosidosis | GM1 Gangliosidosis | Tay-Sachs Disease | Sandhoff Disease | Gaucher Disease, Type 2 | AB Variant Gangliosidosis GM2

United States, Spain, Germany, Italy, Belgium, Brazil, France, Portugal, Switzerland, United Kingdom
IntraBio Inc

Completed

N-Acetyl-L-Leucine for GM2 Gangliosidosis (Tay-Sachs and Sandhoff Disease)

GM2 Gangliosidosis | Tay-Sachs Disease | Sandhoff Disease

United States, Germany, Spain, United Kingdom
Genzyme, a Sanofi Company

Active, not recruiting

A Multinational, Randomized, Double-blind, Placebo-controlled Study to Assess the Efficacy, Pharmacodynamics, Pharmacokinetics, and Safety of Venglustat in Late-onset GM2 (AMETHIST)

Tay-Sachs Disease | Sandhoff Disease

United States, Argentina, Czechia, Portugal, Russian Federation, Turkey, United Kingdom, Brazil, France, Germany, Italy, Japan, Spain
Alexion Pharmaceuticals, Inc.

Recruiting

Evaluation of Pharmacokinetics, Pharmacodynamics, Efficacy, Safety, and Immunogenicity of Ravulizumab Administered Intravenously in Pediatric Participants With Generalized Myasthenia Gravis (gMG)

Generalized Myasthenia Gravis | gMG

Spain, Italy, United States, France, Switzerland, Serbia, Japan, Netherlands

3

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