GM1 GANGLIOSIDOSIS
Clinical trials for GM1 GANGLIOSIDOSIS explained in plain language.
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New pill aims to slow rare childhood brain diseases
Disease control Recruiting nowThis study tests an oral drug called nizubaglustat in children and teens aged 4 and older with rare genetic brain diseases: Niemann-Pick type C, GM1, or GM2 gangliosidosis. The drug is taken by mouth for 18 months and compared to a placebo. The goal is to see if it can safely slo…
Matched conditions: GM1 GANGLIOSIDOSIS
Phase: PHASE3 • Sponsor: Azafaros A.G. • Aim: Disease control
Last updated May 22, 2026 14:04 UTC
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Scientists map rare brain diseases to speed future cures
Knowledge-focused Recruiting nowThis study follows 52 people with Tay-Sachs, Sandhoff, or GM1 gangliosidosis to learn how these rare genetic diseases progress. Researchers measure changes in development, movement, and communication skills each year. No new treatments are given; the goal is to create a clear pic…
Matched conditions: GM1 GANGLIOSIDOSIS
Sponsor: University of Minnesota • Aim: Knowledge-focused
Last updated May 26, 2026 12:08 UTC