One-of-a-kind drug aims to slow rare brain disease in a single child
NCT ID NCT07588581
First seen May 16, 2026 · Last updated May 16, 2026
Summary
This study tests a personalized medicine called an antisense oligonucleotide (ASO) designed for one child with a rare genetic brain disease (CONDBA) that causes brain shrinkage and movement problems. The goal is to see if the drug can improve or stabilize motor skills, coordination, and quality of life over two years. The child must have a confirmed UBTF gene mutation and be able to travel to the study site.
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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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Contacts and locations
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Locations
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Massachusetts General Hospital
Boston, Massachusetts, 02114, United States
Conditions
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