One-Time gene shot aims to halt rare nerve diseases
NCT ID NCT05152823
First seen Nov 01, 2025 · Last updated May 08, 2026 · Updated 24 times
Summary
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 the IGHMBP2 gene directly into the spinal fluid.
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Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
This is a summary of the original study . Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
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Locations
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Nationwide Children's Hospital
Columbus, Ohio, 43205, United States
Conditions
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