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N-lorem Foundation

Clinical trials sponsored by N-lorem Foundation, explained in plain language.

Not yet recruiting 2 Not yet finished but already full! 7 Completed 1

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 18, 2026 12:00 UTC