Breakthrough drug trial aims to halt blindness in rare genetic disease
Disease control
Not yet recruiting
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…
Phase: PHASE3 • Sponsor: Nacuity Pharmaceuticals, Inc. • Aim: Disease control
Last updated Mar 13, 2026 15:06 UTC