One-Person trial aims to halt rare nerve disorder
NCT ID NCT07226297
First seen Nov 10, 2025 · Last updated May 15, 2026 · Updated 24 times
Summary
This study tests a custom-made drug for one person with Charcot-Marie-Tooth disease type 2D, a rare genetic nerve condition that causes muscle weakness. The drug is designed to target the specific genetic error causing the disease. The goal is to see if it can improve motor skills and quality of life over two years.
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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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UTHealth Houston
Houston, Texas, 77030, United States
Conditions
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