GM2 GANGLIOSIDOSIS
Clinical trials for GM2 GANGLIOSIDOSIS explained in plain language.
Never miss a new study
Get alerted when new GM2 GANGLIOSIDOSIS trials appear
Sign up with your email to follow new studies for GM2 GANGLIOSIDOSIS, keep track of the ones that matter, and come back to a personal dashboard instead of checking manually.
By submitting, you agree to our Terms of use
-
New hope for rare brain diseases: long-term drug trial now recruiting
Disease control Recruiting nowThis study tests the long-term safety and effectiveness of a daily medication called nizubaglustat in people with two rare genetic diseases that affect the brain and body. About 21 participants who were in a previous study or who have been on a similar drug (miglustat) will take …
Matched conditions: GM2 GANGLIOSIDOSIS
Phase: PHASE2 • Sponsor: Azafaros B.V. • Aim: Disease control
Last updated Jun 27, 2026 14:00 UTC
-
New pill hopes to tame rare childhood brain diseases
Disease control Recruiting nowThis Phase 3 trial tests an oral drug called nizubaglustat (AZ-3102) in people aged 4 and older with Niemann-Pick type C disease, GM1 gangliosidosis, or GM2 gangliosidosis. The study lasts 18 months and compares the drug to a placebo to see if it is safe and can slow disease prog…
Matched conditions: GM2 GANGLIOSIDOSIS
Phase: PHASE3 • Sponsor: Azafaros B.V. • Aim: Disease control
Last updated Jun 27, 2026 14:00 UTC
-
Scientists track rare brain diseases to pave way for future cures
Knowledge-focused Recruiting nowThis study follows children and adults with Tay-Sachs, Sandhoff, and GM1 gangliosidosis to understand how these diseases progress. Researchers will measure changes in motor skills, communication, and behavior each year. The goal is to create a clear picture of the diseases so fut…
Matched conditions: GM2 GANGLIOSIDOSIS
Sponsor: University of Minnesota • Aim: Knowledge-focused
Last updated Jun 27, 2026 09:10 UTC
-
Massive leukodystrophy biobank aims to unlock disease secrets
Knowledge-focused Recruiting nowThis study collects medical information and biological samples (like blood or tissue) from up to 12,000 people with leukodystrophies—rare disorders that damage the brain's white matter. Researchers will use this data to find new genetic causes, develop biomarkers for future trial…
Matched conditions: GM2 GANGLIOSIDOSIS
Sponsor: Children's Hospital of Philadelphia • Aim: Knowledge-focused
Last updated Jun 27, 2026 07:55 UTC