GM1 GANGLIOSIDOSIS
Clinical trials for GM1 GANGLIOSIDOSIS explained in plain language.
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New pill hopes to tame rare childhood brain diseases
Disease control Recruiting nowThis Phase 3 trial tests an oral drug called nizubaglustat (AZ-3102) in people aged 4 and older with Niemann-Pick type C disease, GM1 gangliosidosis, or GM2 gangliosidosis. The study lasts 18 months and compares the drug to a placebo to see if it is safe and can slow disease prog…
Matched conditions: GM1 GANGLIOSIDOSIS
Phase: PHASE3 • Sponsor: Azafaros B.V. • Aim: Disease control
Last updated Jun 27, 2026 14:00 UTC
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Scientists track rare brain diseases to pave way for future cures
Knowledge-focused Recruiting nowThis study follows children and adults with Tay-Sachs, Sandhoff, and GM1 gangliosidosis to understand how these diseases progress. Researchers will measure changes in motor skills, communication, and behavior each year. The goal is to create a clear picture of the diseases so fut…
Matched conditions: GM1 GANGLIOSIDOSIS
Sponsor: University of Minnesota • Aim: Knowledge-focused
Last updated Jun 27, 2026 09:10 UTC