Important: This page is general information, not medical advice. It does not recommend or prescribe any treatment. Always consult your doctor or pharmacist before starting, stopping or changing any medicine.
About Ataxia Telangiectasia
Ataxia-telangiectasia (A-T) is a rare, inherited disease. It affects the nervous system, immune system, and other body systems. Symptoms appear in young children, usually before age 5. They include:
- Ataxia - trouble coordinating movements
- Poor balance
- Slurred speech
- Tiny, red spider veins, called telangiectasias, on the skin and eyes
- Lung infections
- Delayed physical and sexual development
People with A-T have an increased risk of developing diabetes and cancers, especially lymphoma and leukemia. Although it affects the brain, people with A-T usually have normal or high intelligence.
A-T has no cure. Treatments might improve some symptoms. They include injections to strengthen the immune system, physical and speech therapy, and high-dose vitamins.
It sits within Brain and Nerves and Genetics/Birth Defects.
The lead U.S. institute for this subject is the National Institute of Neurological Disorders and Stroke.
When to speak to someone. Reading about a topic cannot tell you whether it applies to you. Speak to a doctor or pharmacist if symptoms are severe, sudden, getting worse, or simply not going away — and seek emergency care immediately for chest pain, breathing difficulty, sudden weakness or confusion, or a serious allergic reaction.
Research on Ataxia Telangiectasia
59 studies areregistered for this condition on ClinicalTrials.gov, the U.S. National Library of Medicine's public registry. A selection is below — the links go straight to the registry entry, not to a summary of ours.
What a registered study does and does not mean. Registration means a study exists and has been declared publicly. It says nothing about whether the treatment being tested works, is safe, or is available to you. Many studies find no benefit — that is what research is for. Never seek out an experimental treatment on the strength of a registry entry alone.
Research currently under way
- A Study of ART0380 for the Treatment of Advanced or Metastatic Solid TumorsArtios Pharma Ltd
- A Study to Assess the Safety and Tolerability of AZD1390 Given With Radiation Therapy in Patients With Brain CancerAstraZeneca
- Testing the Combination of the Anti-Cancer Drugs Temozolomide and M1774 to Evaluate Their Safety and EffectivenessNational Cancer Institute (NCI)
- Biomarkers for Babies and Young Children With Ataxia TelangiectasiaUniversity of Nottingham
- Tuvusertib Combined With Niraparib or Lartesertib in Participants With Epithelial Ovarian Cancer (DDRiver EOC 302)EMD Serono Research & Development Institute, Inc.
- Tuvusertib (M1774) in Combination With Cemiplimab in Participants With Non-Squamous NSCLC (DDRiver NSCLC 322)EMD Serono Research & Development Institute, Inc.
Research already completed
- Evaluate the Neurological Effects of EryDex on Subjects With A-TQuince Therapeutics S.p.A.
- Tuvusertib (M1774) Human Mass Balance and Absolute Bioavailability Study (DDRIVER Solid Tumors 303)Merck Healthcare KGaA, Darmstadt, Germany, an affiliate of Merck KGaA, Darmstadt, Germany
- A Study Investigating DNA-damage Response Agents in Molecularly Altered Advanced CancerAstraZeneca
Study data from ClinicalTrials.gov, a service of the U.S. National Library of Medicine. Retrieved 2026-07-30. Registry entries are supplied by study sponsors and investigators; ClinicalTrials.gov does not verify their scientific validity. PocketsInfo is not affiliated with, and not endorsed by, the NLM or the NIH.
Where to read more
Rather than paraphrase, we point you to the primary sources. Every link below is to a government health agency, a national institute or a recognised medical body — grouped by what you are trying to find out.
Learn More
- Ataxia - telangiectasiaMedical Encyclopedia
- Ataxia TelangiectasiaNational Institute of Neurological Disorders and Stroke
- Ataxia-Telangiectasia (For Parents)Nemours Foundation
- Learn About A-TAtaxia-Telangiectasia Children's Project
- National Institute of Neurological Disorders and StrokeNational Institute of Neurological Disorders and Stroke
Genetics
- Ataxia-telangiectasia: MedlinePlus GeneticsNational Library of Medicine
Clinical Trials
- ClinicalTrials.gov: Ataxia TelangiectasiaNational Institutes of Health
Medical subject headings
Clinicians and researchers index this subject under Ataxia Telangiectasia. These are the terms to use when searching medical literature.
This page is built from the MedlinePlus health topic record for Ataxia Telangiectasia, a service of the U.S. National Library of Medicine (NLM), National Institutes of Health. Information is from MedlinePlus.gov. PocketsInfo is not affiliated with, and is not endorsed by, the NLM or the NIH. Retrieved 30 July 2026.