Custom drug targets rare genetic brain disease in First-Ever human test
NCT ID NCT06706388
First seen Jun 24, 2026 · Last updated Jun 27, 2026 · Updated 1 time
Summary
This study tests a custom-made drug called an antisense oligonucleotide (ASO) designed for one person with dentatorubral-pallidoluysian atrophy (DRPLA), a rare genetic disorder that affects movement and brain function. The drug aims to reduce the harmful effects of the mutated ATN1 gene. The single participant will be monitored for changes in ataxia (loss of muscle control), seizures, and quality of life over two years.
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-ATN1-002)
What this could lead to
If it works, this could point toward a treatment for DRPLA, a rare and severe genetic brain disorder.
What could go wrong
This is an early-stage, single-participant study, so results may not apply to others. The drug is experimental and risks are unknown.
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.
More trials for these conditions
Other studies related to the condition(s) this trial covers.
Contacts and locations
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Locations
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Columbia University
New York, New York, 10027, United States