GM2 GANGLIOSIDOSIS
Clinical trials for GM2 GANGLIOSIDOSIS explained in plain language.
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New hope for rare brain diseases: Long-Term drug trial launches
Disease control Recruiting nowThis study tests the long-term safety and effects of a daily medication called nizubaglustat in people with two rare genetic brain diseases: GM2 gangliosidosis and Niemann-Pick type C. About 21 participants who were in a previous study or who are already on a similar drug will ta…
Matched conditions: GM2 GANGLIOSIDOSIS
Phase: PHASE2 • Sponsor: Azafaros A.G. • Aim: Disease control
Last updated May 22, 2026 14:04 UTC
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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: GM2 GANGLIOSIDOSIS
Phase: PHASE3 • Sponsor: Azafaros A.G. • Aim: Disease control
Last updated May 22, 2026 14:04 UTC
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Massive leukodystrophy biobank aims to unlock secrets of rare brain diseases
Knowledge-focused Recruiting nowThis study is creating a large collection of medical data and biological samples from people with leukodystrophies—rare disorders that damage the brain's white matter. Researchers will use this information to discover new genetic causes, develop better diagnostic tools, and track…
Matched conditions: GM2 GANGLIOSIDOSIS
Sponsor: Children's Hospital of Philadelphia • Aim: Knowledge-focused
Last updated May 26, 2026 11:05 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: GM2 GANGLIOSIDOSIS
Sponsor: University of Minnesota • Aim: Knowledge-focused
Last updated May 26, 2026 11:03 UTC