Cure My Disease

Don't miss your cure!

Home News Browse About
Log in / Sign up

CACNA1C-related disorder

MONDO:0700321

Any human disease (syndromic or non syndromic) in which the cause of the disease is a variation in the CACNA1C gene.

1 clinical trial for this condition and its sub-types.

Follow this condition — get notified about new trials

Sub-types

Timothy syndrome (1) Timothy syndrome type 1 (1) Brugada syndrome 3 (0) Long QT syndrome 8 (0) Neurodevelopmental disorder with hypotonia, language delay, and skeletal defects with or without seizures (0) Timothy syndrome, atypical type (0) Timothy syndrome, classic type (0) Timothy syndrome type 2 (0)

Broader categories

Disease (618) Hereditary disease (172) Human disease (14) Disease of genetic or genomic mechanism (2) Disease by etiologic mechanism (0)
Not yet recruiting 1
Sort by
  • 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…

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

    Last updated Jun 27, 2026 13:00 UTC

Cure My Disease

Helping patients find clinical trials that match their disease.

Why was Cure my disease built?

Explore

Home News Browse About Terms of use Contact us

This is a site from Cyber and Space