SJOGREN-LARSSON SYNDROME
Clinical trials for SJOGREN-LARSSON SYNDROME explained in plain language.
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New drug shows promise for rare genetic disease
Disease control CompletedThis study tested a new oral drug, ADX-629, in 8 people with Sjögren-Larsson syndrome, a rare inherited disorder that causes lifelong skin, brain, and eye problems due to buildup of harmful fatty aldehydes. The goal was to see if the drug is safe and can reduce these harmful subs…
Matched conditions: SJOGREN-LARSSON SYNDROME
Phase: PHASE1, PHASE2 • Sponsor: University of Nebraska • Aim: Disease control
Last updated May 17, 2026 03:26 UTC
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Gene sequencing breakthrough could unlock mysteries 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 doctors more accurately diagnose children with leukodystrophies—rare diseases that damage the brain's white matter. Researchers enrolled 236 children and their parents to see if this…
Matched conditions: SJOGREN-LARSSON SYNDROME
Sponsor: Children's Hospital of Philadelphia • Aim: Knowledge-focused
Last updated May 14, 2026 12:04 UTC