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A Natural History Study of the Gangliosidoses
Hypothesis: To characterize and describe disease progression and heterogeneity of the gangliosidosis diseases. This research study seeks to develop a quantita…
Tay-Sachs DiseaseSandhoff DiseaseLate Onset Tay-Sachs Disease+2 more
University of MinnesotaNCT00668187
FLOWER: Following Longitudinal Outcomes With Epidemiology for Rare Diseases
FLOWER is a completely virtual, nationwide, real-world observational study to collect, annotate, standardize, and report clinical data for rare diseases. Patie…
Alpha-ThalassemiaBeta-ThalassemiaAmyloidosis+14 more
xCuresNCT06539169
Phase 3
18-month Double-blind, Randomized, Placebo-controlled, Multicenter, Phase 3 Study to Evaluate the Safety and Efficacy of Oral Nizubaglustat (AZ-3102) in Late-infantile and Juvenile Forms of Niemann-Pick Type C Disease and in Late-infantile and Juvenile-onset Forms of GM1 Gangliosidosis or GM2 Gangliosidosis
An 18-month double-blind, randomized, placebo-controlled, multicenter, Phase 3 study to evaluate the safety and efficacy of oral nizubaglustat (AZ-3102) in lat…
Niemann-Pick Type C DiseaseGM1 GangliosidosisGM2 Gangliosidosis
Azafaros A.G.NCT07054515