GM1 GANGLIOSIDOSIS
Clinical trials for GM1 GANGLIOSIDOSIS explained in plain language.
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New pill aims to slow rare brain diseases in kids
Disease control Recruiting nowThis study tests an oral drug called nizubaglustat in children aged 4 and older with Niemann-Pick type C disease or GM1/GM2 gangliosidosis. The goal is to see if the drug can safely slow the progression of these rare, serious brain disorders. Participants will take the drug or a …
Matched conditions: GM1 GANGLIOSIDOSIS
Phase: PHASE3 • Sponsor: Azafaros A.G. • Aim: Disease control
Last updated May 13, 2026 15:59 UTC
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New study tracks rare brain diseases to pave way for future cures
Knowledge-focused Recruiting nowThis study follows 52 people with Tay-Sachs, Sandhoff, or GM1 gangliosidosis to understand how these rare genetic diseases progress. Researchers measure changes in motor skills, communication, and behavior each year. The goal is to create a clear picture of the diseases so future…
Matched conditions: GM1 GANGLIOSIDOSIS
Sponsor: University of Minnesota • Aim: Knowledge-focused
Last updated May 11, 2026 20:39 UTC