Spinal injection gene therapy trial offers hope for devastating childhood diseases
Disease control
ENROLLING_BY_INVITATION
This early-phase trial is testing a one-time spinal injection of a gene therapy for children with rare, life-limiting neuromuscular diseases called SMARD1 and CMT2S. The goal is to deliver a working copy of a missing gene to slow or stop disease progression. The study will first …
Phase: PHASE1, PHASE2 • Sponsor: Megan Waldrop • Aim: Disease control
Last updated Mar 27, 2026 12:39 UTC