Custom-Made genetic drug tested in single ALS patient

NCT ID NCT07423494

First seen Jun 27, 2026 · Last updated Jun 27, 2026

Summary

This study tests a custom-made drug designed for one person with ALS caused by a specific gene mutation (CHCHD10). The drug aims to slow disease progression by targeting the genetic root cause. The participant will be closely monitored for changes in muscle strength, breathing, and thinking over 12 months.

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Conditions

The condition(s) this trial relates to.

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

Contacts and locations

Locations

  • Mayo Clinic

    Rochester, Minnesota, 55905, United States

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