Experimental drug targets genetic cause of alport syndrome in tiny pilot
Disease control
Completed
This phase 2 pilot study tested a drug called ELX-02 in 3 adults with Alport syndrome caused by specific 'nonsense' mutations. The goal was to see if the drug is safe and if it can reduce protein in the urine, a sign of kidney damage. Participants received daily injections for 8 …
Phase: PHASE2 • Sponsor: Eloxx Pharmaceuticals, Inc. • Aim: Disease control
Last updated Jun 27, 2026 09:07 UTC