Custom-Made genetic drug targets rare brain disorder in one patient
NCT ID NCT06816498
First seen Jul 13, 2026 · Last updated Jul 14, 2026 · Updated 1 time
Summary
This study tests a personalized medicine called an antisense oligonucleotide, designed specifically for one person with autosomal dominant leukodystrophy (ADLD), a rare genetic brain disease. The treatment aims to slow or stop the disease by targeting the underlying genetic mutation. The participant will be followed for two years to measure changes in gait, neurological function, and brain structure.
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-LMNB1-001)
- What this could lead to
- If successful, this approach could slow or halt the progression of a rare, debilitating neurological disease and pave the way for similar personalized treatments for other genetic disorders.
- What could go wrong
- This is a very early, single-participant study, so results may not apply to others. The treatment is experimental and carries unknown risks, including potential side effects from the antisense oligonucleotide.
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
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Locations
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Mayo Clinic
Rochester, Minnesota, 55905, United States