Hope for rare brain disease: Final-Stage trial tests promising pill
Disease control
Not yet recruiting
This study is testing if a medication called N-Acetyl-L-Leucine can help control symptoms and improve daily life for people with rare, inherited disorders caused by a faulty CACNA1A gene. These disorders affect coordination, balance, and speech. In this final-stage trial, 60 part…
Phase: PHASE3 • Sponsor: IntraBio Inc • Aim: Disease control
Last updated Mar 27, 2026 12:40 UTC