Custom drug targets rare ALS in first-of-its-kind single-patient trial
NCT ID NCT07423494
First seen Feb 21, 2026 · Last updated May 16, 2026 · Updated 10 times
Summary
This study tests a custom-made antisense oligonucleotide (ASO) designed for one person with ALS caused by a specific CHCHD10 gene mutation. The goal is to slow disease progression by targeting the genetic root cause. The single participant will receive the drug and be monitored for changes in muscle strength, breathing, and thinking over 12 months.
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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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Locations
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Mayo Clinic
Rochester, Minnesota, 55905, United States
Conditions
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