Gm1 Gangliosidosis
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CLINICAL_TRIALS trial_result · 2025-10-24Clinical Trial Update: Natural History Study of Infantile and Juvenile GM1 Gangliosidosis (GM1) Patients
This clinical trial update reports that the natural history study of GM1 Gangliosidosis in infantile and juvenile patients has completed. It…
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CLINICAL_TRIALS trial_result · 2025-09-29Clinical Trial Update: GM1 and GM2 Gangliosidosis PROspective Neurological Disease TrajectOry Study (PRONTO)
A clinical trial update for the PROspective Neurological Disease Trajectory Study (PRONTO) by Azafaros reports completion of the GM1 and GM2…
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CLINICAL_TRIALS trial_result · 2021-04-14Clinical Trial Update: Synergistic Enteral Regimen for Treatment of the Gangliosidoses [Phase 4]
Phase 4 trial update for a synergistic enteral regimen in GM1 and GM2 gangliosidoses; the study was terminated with results posted in 2021.