Friedreich ataxia
MONDO:0100339An inherited condition that affects the nervous system and causes movement problems. People with this condition develop impaired muscle coordination (ataxia) that worsens over time. Other features include the gradual loss of strength and sensation in the arms and legs, muscle stiffness (spasticity), and impaired speech. Many individuals have a form of heart disease called hypertrophic cardiomyopathy. Some develop diabetes, impaired vision, hearing loss, or an abnormal curvature of the spine (scoliosis). Most people with Friedreich ataxia begin to experience the signs and symptoms around puberty.
Also known as: FA, FRDA, Friedreich ataxia, Friedreich's Ataxia, Friedreich's ataxia, Friedreich ataxia with retained reflexes, hereditary spinal ataxia, hereditary spinal sclerosis
55 clinical trials for this condition and its sub-types, 37 tagged with Friedreich ataxia itself.
Follow this condition to get notified about new trialsWhere it sits in the disease tree
Browse by category →Sub-types of Friedreich ataxia
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Friedreich ataxia 1 1 trial
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Friedreich ataxia 2 0 trials
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Children's trial launches for Friedreich's ataxia drug
Disease control OngoingThis study tests omaveloxolone, a drug already approved for adults with Friedreich's ataxia, in children aged 2 to 15. Researchers want to see how the drug moves through children's bodies and whether it is safe. The study involves two parts: a single dose followed by daily dosing…
Phase 1 • Sponsor: Biogen • Aim: Disease control
Last updated Sep 05, 2026 00:00 UTC
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Gene therapy trial aims to repair hearts in rare neurological disease
Disease control OngoingThis early-stage trial tests a gene therapy called LX2006 in 8 people with Friedreich's ataxia who also have heart muscle disease. The therapy delivers a healthy copy of the FXN gene to heart cells using a modified virus. The main goal is to check safety over 5 years, while also …
Phase 1/2 • Sponsor: Lexeo Therapeutics • Aim: Disease control
Last updated Jun 27, 2026 08:06 UTC
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Gene therapy trial for rare heart condition halted before it began
Disease control CancelledThis study was designed to test a new gene therapy called ASP2016 for heart problems caused by Friedreich Ataxia, a rare genetic disease. The therapy aimed to deliver a healthy copy of the faulty gene to help the heart. However, the study was withdrawn before any participants wer…
Phase 1 • Sponsor: Astellas Gene Therapies • Aim: Disease control
Last updated Jun 27, 2026 08:00 UTC
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Can a new drug slow friedreich ataxia? Long-Term trial underway
Disease control OngoingThis study is testing the long-term safety of a drug called vatiquinone in 130 people with Friedreich ataxia, a rare genetic disease that affects movement and coordination. All participants have taken vatiquinone before in earlier studies. Researchers will track side effects and …
Phase 3 • Sponsor: PTC Therapeutics • Aim: Disease control
Last updated Jun 27, 2026 07:56 UTC
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New test could predict falls in muscle disease patients
Diagnosis By invitation onlyThis study aims to create a simple test battery to determine fall risk in people with neuromuscular disorders, such as muscular dystrophy or ALS. Researchers will assess 108 participants using several physical tests like walking, standing, and rising from a chair. The goal is to …
Sponsor: LMU Klinikum • Aim: Diagnosis
Last updated Jun 26, 2026 16:30 UTC
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Brain function in Friedreich's ataxia: new clues from genetic testing
Knowledge-focused OngoingThis study looks at how Friedreich's ataxia, a genetic nerve disease, affects thinking skills like memory and problem-solving. Researchers will compare cognitive test results between people with the condition and healthy volunteers, and see if these results relate to the number o…
Sponsor: Institut National de la Santé Et de la Recherche Médicale, France • Aim: Knowledge-focused
Last updated Jul 23, 2026 00:00 UTC
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New brain scans could reveal hidden clues in rare nerve disease
Knowledge-focused By invitation onlyThis study uses special brain scans (MRS) to measure two natural chemicals, GABA and glutathione, in people with Friedreich's Ataxia (FRDA). Researchers want to see if these chemicals can serve as reliable markers of the disease and how they change after taking the drug Omaveloxo…
Sponsor: Children's Hospital of Philadelphia • Aim: Knowledge-focused
Last updated Jun 27, 2026 13:05 UTC