First drug trial targets root cause of rare genetic disorder
NCT ID NCT05443685
Summary
This early-stage study is testing whether a new oral medication called ADX-629 is safe and can reduce the buildup of harmful chemicals in people with Sjögren-Larsson syndrome (SLS), a rare genetic disorder. Up to 8 participants with SLS will take the drug for 12 weeks. Researchers will check for safety and measure if the drug improves the underlying chemical problems that cause skin, brain, and eye symptoms.
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Contacts and locations
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Locations
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University of Nebraska Medical Center
Omaha, Nebraska, 68198, United States
Conditions
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