GM2 GANGLIOSIDOSIS
Clinical trials for GM2 GANGLIOSIDOSIS explained in plain language.
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New drug offers hope for families battling rare, devastating brain diseases
Disease control Recruiting nowThis study aims to gather long-term safety and effectiveness data for the drug nizubaglustat in people with rare, progressive brain disorders (GM2 gangliosidosis or Niemann-Pick type C). It includes two groups: patients continuing from a previous study and patients switching from…
Matched conditions: GM2 GANGLIOSIDOSIS
Phase: PHASE2 • Sponsor: Azafaros A.G. • Aim: Disease control
Last updated Mar 27, 2026 12:39 UTC
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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: GM2 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: GM2 GANGLIOSIDOSIS
Sponsor: University of Minnesota • Aim: Knowledge-focused
Last updated Mar 30, 2026 14:34 UTC
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Massive global hunt for answers to devastating brain diseases
Knowledge-focused Recruiting nowThis study aims to collect medical information and biological samples from 12,000 people worldwide with rare genetic brain disorders called leukodystrophies. Researchers will use this data to better understand these diseases, improve diagnosis, and identify targets for future tre…
Matched conditions: GM2 GANGLIOSIDOSIS
Sponsor: Children's Hospital of Philadelphia • Aim: Knowledge-focused
Last updated Mar 25, 2026 14:09 UTC