GM2 GANGLIOSIDOSIS
Clinical trials for GM2 GANGLIOSIDOSIS explained in plain language.
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New pill shows promise for rare genetic brain disorders
Disease control CompletedThis study tested an experimental oral drug, AZ-3102, in 13 people aged 12 and older with GM2 gangliosidosis (Tay-Sachs or Sandhoff disease) or Niemann-Pick type C disease. The main goals were to check the drug's safety and how it moves through the body over 12 weeks. Participant…
Matched conditions: GM2 GANGLIOSIDOSIS
Phase: PHASE2 • Sponsor: Azafaros A.G. • Aim: Disease control
Last updated May 26, 2026 12:13 UTC
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Small study maps rare Tay-Sachs progression to speed future treatments
Knowledge-focused CompletedThis study followed 10 people with late onset Tay-Sachs disease for 6 months to learn how the condition changes over time. Researchers measured balance, coordination, and brain chemicals. The goal was to gather information that will help design better clinical trials for future t…
Matched conditions: GM2 GANGLIOSIDOSIS
Sponsor: Massachusetts General Hospital • Aim: Knowledge-focused
Last updated May 22, 2026 13:54 UTC
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Gene sequencing breakthrough may unlock mysterious brain diseases in kids
Knowledge-focused CompletedThis study looked at whether whole genome sequencing (a complete scan of a person's DNA) can help doctors diagnose leukodystrophies—rare brain disorders that damage white matter—more accurately than standard methods. About 236 children with abnormal brain MRI scans but no known g…
Matched conditions: GM2 GANGLIOSIDOSIS
Sponsor: Children's Hospital of Philadelphia • Aim: Knowledge-focused
Last updated May 18, 2026 12:01 UTC