One-of-a-kind drug targets rare ALS gene in first human test
NCT ID NCT07423494
First seen Feb 21, 2026 · Last updated May 21, 2026 · Updated 12 times
Summary
This study tests a personalized medicine designed for one person with ALS caused by a specific CHCHD10 gene mutation. The drug is an antisense oligonucleotide (ASO) that aims to slow disease progression. The single participant will be monitored for changes in muscle strength, breathing, and thinking over 12 months. This early-phase trial focuses on safety and how well the drug works for this individual.
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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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Mayo Clinic
Rochester, Minnesota, 55905, United States
Conditions
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