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Results for “Sandhoffs Disease”

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Showing 5 of 0 results

Large-scale testing (Phase 3)Ended earlyNCT04221451
What this trial is testing

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

Who this might be right for
Tay-Sachs DiseaseSandhoff Disease
Genzyme, a Sanofi Company 75
Not applicableStudy completedNCT01869270
What this trial is testing

Gene Therapy for Tay-Sachs Disease

Who this might be right for
Tay Sachs DiseaseSandhoff DiseaseLate Onset Tay Sachs Disease
University of Minnesota 4
Not applicableNot Yet RecruitingNCT07445490
What this trial is testing

Translational Potential of ex Vivo Gene Therapy in GM2 Gangliosidosis

Who this might be right for
Tay-Sachs Disease GangliosideSandhoff Disease Ganglioside
Assistance Publique - Hôpitaux de Paris 6
Not applicableLooking for participantsNCT00668187
What this trial is testing

A Natural History Study of the Gangliosidoses

Who this might be right for
Tay-Sachs DiseaseSandhoff DiseaseLate Onset Tay-Sachs Disease+2 more
University of Minnesota 52
Early research (Phase 1)Looking for participantsNCT02254863
What this trial is testing

UCB Transplant of Inherited Metabolic Diseases With Administration of Intrathecal UCB Derived Oligodendrocyte-Like Cells

Who this might be right for
AdrenoleukodystrophyBatten DiseaseMucopolysaccharidosis II+9 more
Joanne Kurtzberg, MD 40