Custom Gene-Targeting drug tested in single child with rare brain disease
NCT ID NCT07410143
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 child with a rare, severe genetic brain disorder. The drug aims to correct the specific genetic mistake causing the disease. The trial focuses on safety and how the drug moves through the body, with only one participant enrolled.
What this could mean
Our plain-language read of the trial. This is informational only — not medical advice or a prediction.
Active substance
individualized antisense oligonucleotide (ASO)
What this could lead to
If successful, this approach could point toward a way to treat rare genetic brain diseases by correcting the specific genetic error in each patient.
What could go wrong
This is a very early trial with only 1 participant, so results may not apply to others. The treatment is experimental and may cause side effects or not work at all.
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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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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Great Ormond Street Hospital
London, United Kingdom