New drug shows promise for rare genetic disease
NCT ID NCT05443685
First seen Feb 16, 2026 · Last updated May 15, 2026 · Updated 11 times
Summary
This 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 substances. Participants took the drug for 12 weeks and were monitored for side effects and changes in disease markers.
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Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
This is a summary of the original study . Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
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Locations
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University of Nebraska Medical Center
Omaha, Nebraska, 68198, United States
Conditions
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