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Timothy syndrome, classic type

MONDO:0021171

Classic form of Timothy syndrome, includes all features of generic.

Also known as: Timothy syndrome type 1 (disorder)

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

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Sub-types

Timothy syndrome type 1 (1) Timothy syndrome type 2 (0)

Broader categories

Disease (618) Hereditary disease (172) Syndromic disease (24) Long QT syndrome (18) Human disease (14) Disease of genetic or genomic mechanism (2) Timothy syndrome (1) Autosomal dominant disease (0) Autosomal genetic disease (0) CACNA1C-related disorder (0)

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…

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

Last updated Jun 27, 2026 13:00 UTC