One-Time gene shot aims to halt rare nerve diseases
Disease control
ENROLLING_BY_INVITATION
This early-stage trial tests a single injection of gene therapy for two rare nerve diseases, SMARD1 and CMT2S. Ten participants, including children who cannot walk, will receive the treatment to see if it is safe and can improve movement. The therapy delivers a working copy of th…
Phase: PHASE1, PHASE2 • Sponsor: Megan Waldrop • Aim: Disease control
Last updated May 11, 2026 20:48 UTC