Custom drug targets rare genetic ALS in First-Ever Single-Patient trial
NCT ID NCT07095712
First seen Apr 12, 2026 · Last updated May 16, 2026 · Updated 9 times
Summary
This study tested a custom-made drug designed for one person with ALS caused by a specific genetic change in the TARDBP gene. The drug aims to slow disease progression by targeting the genetic root of the condition. The participant's muscle strength, breathing, and thinking skills were tracked for 12 months to see if the treatment helped.
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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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Columbia University, Irving Medical Center
New York, New York, 10032, United States
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Houston Methodist
Houston, Texas, 77030, United States
Conditions
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