Could a common cholesterol drug slow batten disease?
Disease control
Not yet recruiting
This phase 3 trial tests whether PLX-200 (gemfibrozil) can safely slow the progression of CLN3 disease, a rare genetic disorder that causes loss of motor skills, vision, and seizures in children. About 39 kids aged 6-18 will receive either the drug or a placebo for up to 96 weeks…
Phase: PHASE3 • Sponsor: Polaryx Therapeutics, Inc. • Aim: Disease control
Last updated Jun 27, 2026 12:30 UTC