Custom gene-targeting drug tested in single patient with rare nerve disease
NCT ID NCT07223632
First seen Nov 03, 2025 · Last updated May 13, 2026 · Updated 29 times
Summary
This study tests a custom-made drug (VCA-894A) in one person with a specific genetic form of Charcot-Marie-Tooth disease (CMT2S). The drug is designed to fix a genetic error and restore a missing protein. It is given via spinal injection to see if it is safe and can improve muscle function.
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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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Vanda Investigational Site
Madison, Wisconsin, 53792, United States
Conditions
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