SMARD1

Clinical trials for SMARD1 explained in plain language.

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Not yet finished but already full! 1

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…

Matched conditions: SMARD1

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

Last updated May 11, 2026 20:48 UTC

SMARD1

Get alerted when new SMARD1 trials appear

One-Time gene shot aims to halt rare nerve diseases