GM1 GANGLIOSIDOSIS
Clinical trials for GM1 GANGLIOSIDOSIS explained in plain language.
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New hope for kids with devastating rare brain diseases
Disease control Recruiting nowThis study is testing whether an oral drug called nizubaglustat can safely slow down or control the progression of several rare, inherited brain diseases in children. It will involve about 147 participants, aged 4 and older, who have conditions like Niemann-Pick Type C or GM1/GM2…
Matched conditions: GM1 GANGLIOSIDOSIS
Phase: PHASE3 • Sponsor: Azafaros A.G. • Aim: Disease control
Last updated Mar 27, 2026 12:38 UTC
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Scientists map the devastating course of rare childhood diseases
Knowledge-focused Recruiting nowThis study aims to carefully track how rare, inherited disorders called gangliosidoses (like Tay-Sachs and Sandhoff disease) progress over time. Researchers will follow about 52 participants, from infants to adults, using tests and brain scans to measure changes in development, m…
Matched conditions: GM1 GANGLIOSIDOSIS
Sponsor: University of Minnesota • Aim: Knowledge-focused
Last updated Mar 30, 2026 14:34 UTC