One-Person trial aims to treat rare nerve disorder with custom drug
NCT ID NCT07226297
First seen Jun 27, 2026 · Last updated Jun 27, 2026
Summary
This study tests a custom-made drug called an antisense oligonucleotide (ASO) designed for one person with Charcot-Marie-Tooth disease type 2D (CMT2D) caused by a specific GARS1 gene mutation. The drug aims to improve motor skills and quality of life. Only one participant is enrolled, and the study will measure changes over 24 months.
What this could mean
Our plain-language read of the trial. This is informational only — not medical advice or a prediction.
Active substance
personalized antisense oligonucleotide (nL-GARS1-001)
What this could lead to
If it works, this could point toward a treatment for CMT2D caused by specific GARS1 mutations.
What could go wrong
This is a very early, single-participant study, so results may not apply to others. The treatment is experimental and may not improve symptoms or could cause side effects.
Disclaimer
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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.
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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Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
Contacts and locations
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Locations
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UTHealth Houston
Houston, Texas, 77030, United States