CACNA1C

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Trials to join now! 1 Not yet recruiting 1

First human trial of TS1-ASO aims to tame rare genetic brain disorder

Disease control Not yet recruiting

This first-in-human trial tests a genetic drug called TS1-ASO in 5 children with Timothy Syndrome, a rare condition causing severe heart and brain problems. The drug is injected into the spine to target the genetic root of the disease. The main goal is to check safety and how the…

Matched conditions: CACNA1C

Phase: PHASE1, PHASE2 • Sponsor: Stanford University • Aim: Disease control

Last updated Jun 27, 2026 13:00 UTC

CACNA1C

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First human trial of TS1-ASO aims to tame rare genetic brain disorder