Custom-Made drug targets rare brain disease in one child
NCT ID NCT07588581
First seen May 16, 2026 · Last updated May 18, 2026 · Updated 1 time
Summary
This study tests a custom-made genetic medicine (called an antisense oligonucleotide) designed specifically for one child with a rare, inherited brain disease that causes brain shrinkage and loss of movement. The goal is to see if the drug can slow or improve motor skills and quality of life over two years. Only one participant is enrolled, and the treatment is personalized to their unique genetic mutation.
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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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Genom att skicka in godkänner du våra Användarvillkor
Contacts and locations
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Genom att skicka in godkänner du våra Användarvillkor
Locations
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Massachusetts General Hospital
Boston, Massachusetts, 02114, United States
Conditions
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