CMT2S

Clinical trials for CMT2S explained in plain language.

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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 …

Matched conditions: CMT2S

Phase: PHASE1, PHASE2 • Sponsor: Megan Waldrop • Aim: Disease control

Last updated Mar 27, 2026 12:39 UTC

CMT2S

Get alerted when new CMT2S trials appear

Spinal injection gene therapy trial offers hope for devastating childhood diseases