GM2 GANGLIOSIDOSIS
Clinical trials for GM2 GANGLIOSIDOSIS explained in plain language.
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New hope for rare brain diseases: long-term safety trial of nizubaglustat begins
Disease control Recruiting nowThis study tests the long-term safety and effects of a daily drug called nizubaglustat in people with two rare genetic diseases that affect the brain: GM2 gangliosidosis and Niemann-Pick type C. About 21 participants who were in an earlier study or who are currently on another dr…
Matched conditions: GM2 GANGLIOSIDOSIS
Phase: PHASE2 • Sponsor: Azafaros A.G. • Aim: Disease control
Last updated May 15, 2026 11:57 UTC
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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: GM2 GANGLIOSIDOSIS
Phase: PHASE3 • Sponsor: Azafaros A.G. • Aim: Disease control
Last updated May 13, 2026 15:59 UTC
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Massive leukodystrophy biobank aims to unlock disease secrets
Knowledge-focused Recruiting nowThis study collects medical information and biological samples from up to 12,000 people with leukodystrophies—rare brain disorders affecting white matter. Researchers will use this data to find new genetic causes, develop biomarkers, and better understand how these diseases progr…
Matched conditions: GM2 GANGLIOSIDOSIS
Sponsor: Children's Hospital of Philadelphia • Aim: Knowledge-focused
Last updated May 13, 2026 16:02 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: GM2 GANGLIOSIDOSIS
Sponsor: University of Minnesota • Aim: Knowledge-focused
Last updated May 11, 2026 20:39 UTC