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.
This is an AI summary of the original study and may miss details. Read our disclaimer.
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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
Locations
-
UTHealth Houston
Houston, Texas, 77030, United States