Breakthrough drug trial aims to halt blindness in rare genetic disease
NCT ID NCT07290530
Summary
This study is testing whether a new drug called NPI-001 can slow down vision loss in adults with Usher syndrome, a rare genetic condition that causes both hearing and vision problems. Over 24 months, 80 participants will take either NPI-001 or a placebo (inactive substance) twice daily to see if the drug helps preserve the light-sensing cells in the eye. Researchers will monitor vision changes and check for any side effects.
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Conditions
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