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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Precision genetic therapy aims to slow rare brain disease
Disease control Recruiting nowThis early-stage trial tests a drug called atipeksen for people with Ataxia-Telangiectasia (A-T), a rare genetic disease that affects movement and coordination. The drug is designed to fix a specific genetic error in the ATM gene and is given via spinal injection. The study will …
Phase 1/2 • Sponsor: Timothy Yu • Aim: Disease control
Last updated Jun 27, 2026 12:38 UTC
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New hope for rare disease: expanded access to experimental drug for A-T patients
Disease control Expanded accessThis program provides an experimental drug, levacetylleucine, to people with Ataxia-Telangiectasia (A-T), a rare genetic disease that affects movement and immunity. It is for patients who cannot join clinical trials and have no other good treatment options. The goal is to offer p…
Sponsor: IntraBio Inc • Aim: Disease control
Last updated Jun 27, 2026 09:07 UTC
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New drug cocktail aims to shrink tough cancers
Disease control Recruiting nowThis study tests a new drug called TCC1727 combined with other medicines (benmelstobart, olaparib, or topotecan) in people with advanced solid tumors, including lung, stomach, endometrial cancer, and melanoma. The goal is to find the safest dose and see if the combination helps s…
Phase 1/2 • Sponsor: Beijing Tide Pharmaceutical Co., Ltd • Aim: Disease control
Last updated Jun 27, 2026 09:00 UTC