SJOGREN-LARSSON SYNDROME
Clinical trials for SJOGREN-LARSSON SYNDROME explained in plain language.
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Experimental pill targets toxic buildup in rare genetic disease
Disease control CompletedThis study tested an oral drug called ADX-629 in 8 people with Sjögren-Larsson syndrome, a rare inherited condition that causes lifelong skin, brain, and eye problems due to harmful fat buildup. The goal was to see if the drug is safe and can reduce these toxic substances. Partic…
Matched conditions: SJOGREN-LARSSON SYNDROME
Phase: PHASE1, PHASE2 • Sponsor: University of Nebraska • Aim: Disease control
Last updated May 26, 2026 11:46 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: SJOGREN-LARSSON SYNDROME
Sponsor: Children's Hospital of Philadelphia • Aim: Knowledge-focused
Last updated May 18, 2026 12:01 UTC