Custom drug targets rare genetic ALS in First-Ever Single-Patient trial
Disease control
Completed
This study tested a custom-made drug designed for one person with ALS caused by a specific genetic change in the TARDBP gene. The drug aims to slow disease progression by targeting the genetic root of the condition. The participant's muscle strength, breathing, and thinking skill…
Phase: PHASE1, PHASE2 • Sponsor: n-Lorem Foundation • Aim: Disease control
Last updated May 17, 2026 11:08 UTC