SJOGREN-LARSSON SYNDROME
Clinical trials for SJOGREN-LARSSON SYNDROME explained in plain language.
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Experimental pill targets toxic fat 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 disorder that causes harmful fatty substances to build up in the skin, brain, and eyes. The main goals were to see if the drug is safe and can reduce these fatty aldehydes. P…
Matched conditions: SJOGREN-LARSSON SYNDROME
Phase: PHASE1, PHASE2 • Sponsor: University of Nebraska • Aim: Disease control
Last updated Jun 27, 2026 12:29 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: SJOGREN-LARSSON SYNDROME
Sponsor: Children's Hospital of Philadelphia • Aim: Knowledge-focused
Last updated Jun 27, 2026 07:56 UTC