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2 Studies found from all possible combinations of your search terms. Select a protocol that you wish to review.
Protocol Number Title Protocol Status Min-Max Age Institute Keywords
20-HG-0156A 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) No longer recruiting/follow-up only2-125 YearsNHGRILate-Onset
11-EI-0245Generation of Induced Pluripotent Stem (iPS) Cell Lines from Somatic Cells of Participants with Eye Diseases and from Somatic Cells of Matched ControlsParticipants currently recruited/enrolled1-125 YearsNEI Late-Onset