GM2 GANGLIOSIDOSIS
Clinical trials for GM2 GANGLIOSIDOSIS explained in plain language.
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New pill shows promise for rare brain disorders in early trial
Disease control CompletedThis study tested an oral drug called AZ-3102 in 13 people with GM2 gangliosidosis (Tay-Sachs or Sandhoff disease) or Niemann-Pick type C disease. The main goal was to check safety and how the drug moves through the body over 12 weeks. It was a randomized, double-blind, placebo-c…
Matched conditions: GM2 GANGLIOSIDOSIS
Phase: PHASE2 • Sponsor: Azafaros B.V. • Aim: Disease control
Last updated Jun 27, 2026 12:33 UTC
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Small study tracks rare disease to pave way for future treatments
Knowledge-focused CompletedThis study followed 10 people with late onset Tay-Sachs disease to see how their symptoms and body chemistry changed over six months. Researchers measured balance, coordination, and brain chemicals. The goal was to gather information that will help design better clinical trials f…
Matched conditions: GM2 GANGLIOSIDOSIS
Sponsor: Massachusetts General Hospital • Aim: Knowledge-focused
Last updated Jun 27, 2026 09:09 UTC
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Could a single DNA test solve the mystery of rare brain diseases in kids?
Knowledge-focused CompletedThis study looked at whether whole genome sequencing (a complete read of a person's DNA) can help diagnose leukodystrophies, a group of rare brain diseases that are hard to identify. Researchers enrolled 236 children with white matter abnormalities on brain scans but no known gen…
Matched conditions: GM2 GANGLIOSIDOSIS
Sponsor: Children's Hospital of Philadelphia • Aim: Knowledge-focused
Last updated Jun 27, 2026 07:56 UTC