Ataxia telangiectasia
MONDO:0008840Ataxia-telangiectasia is the association of severe combined immunodeficiency (affecting mainly the humoral immune response) with progressive cerebellar ataxia. It is characterized by neurological signs, telangiectasias, increased susceptibility to infections and a higher risk of cancer.
Also known as: Louis-Bar syndrome, ataxia - telangiectasia, ataxia telangiectasia, ataxia telangiectasia syndrome, AT, AT, complementation group A, AT, complementation group C, AT, complementation group D
35 clinical trials for this condition and its sub-types, 11 tagged with Ataxia telangiectasia itself.
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Sub-types of Ataxia telangiectasia
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Continued EryDex treatment studied in rare neurological disorder
Disease control Stopped earlyThis study offered continued treatment with EryDex to 101 people with ataxia telangiectasia (A-T) who had finished a previous trial. The main goal was to monitor safety, including side effects and serious events. The study was terminated early, and it did not aim to cure the dise…
Phase 3 • Sponsor: Quince Therapeutics S.p.A. • Aim: Disease control
Last updated Jun 27, 2026 12:29 UTC
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Experimental drug shows promise for rare genetic disorder
Disease control Stopped earlyThis Phase II trial tested a drug called N-Acetyl-L-Leucine (IB1001) in 17 people with Ataxia-Telangiectasia, a rare genetic disease that affects movement and immunity. The study aimed to see if the drug could improve symptoms and slow the disease over time. The trial was termina…
Phase 2 • Sponsor: IntraBio Inc • Aim: Disease control
Last updated Jun 27, 2026 12:00 UTC