FRIEDREICH ATAXIA
Clinical trials for FRIEDREICH ATAXIA explained in plain language.
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Gene therapy aims to fix heart damage in rare nerve disease
Disease control Recruiting nowThis early-stage study tests a gene therapy called AAVrh.10hFXN for heart problems caused by Friedreich's ataxia, a rare genetic disorder. The therapy delivers a working gene to help heart cells function better. About 25 people aged 12 to 50 with mild to moderate heart disease wi…
Matched conditions: FRIEDREICH ATAXIA
Phase: PHASE1 • Sponsor: Weill Medical College of Cornell University • Aim: Disease control
Last updated May 26, 2026 08:53 UTC
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Hope for Friedreich's ataxia: new drug trial seeks Long-Term answers
Disease control Recruiting nowThis study is testing the long-term safety and effects of an experimental drug called CTI-1601 in people with Friedreich's ataxia, a rare genetic disease that affects movement and heart function. It includes about 85 participants aged 2 and older, some of whom have taken the drug…
Matched conditions: FRIEDREICH ATAXIA
Phase: PHASE2 • Sponsor: Larimar Therapeutics, Inc. • Aim: Disease control
Last updated May 26, 2026 08:41 UTC
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Hope for young Friedreich's patients: drug approved for adults now tested in kids
Disease control Recruiting nowThis study tests omaveloxolone (SKYCLARYS), a drug already approved for adults with Friedreich's ataxia, in children aged 2 to 15. The goal is to see if it improves balance and stability and is safe for younger patients. About 255 participants will receive either the drug or a pl…
Matched conditions: FRIEDREICH ATAXIA
Phase: PHASE3 • Sponsor: Biogen • Aim: Disease control
Last updated May 26, 2026 08:32 UTC
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New hope for swallowing difficulties in rare genetic disease
Symptom relief Recruiting nowThis study is testing whether the drug omaveloxolone can improve swallowing difficulties in people with Friedreich's ataxia, a rare nerve disease that affects movement and coordination. About 40 French patients who have been taking the drug for at least six months will fill out a…
Matched conditions: FRIEDREICH ATAXIA
Sponsor: Centre Hospitalier Universitaire de Nice • Aim: Symptom relief
Last updated May 26, 2026 08:30 UTC
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New study uses smartphone app to track Real-Life impact of friedreich ataxia
Knowledge-focused Recruiting nowThis study observes 200 people with Friedreich Ataxia in Germany, Austria, and France to understand their daily challenges, quality of life, and healthcare costs. Participants use a mobile app for six months to report symptoms and experiences. The goal is to gather insights, not …
Matched conditions: FRIEDREICH ATAXIA
Sponsor: German Center for Neurodegenerative Diseases (DZNE) • Aim: Knowledge-focused
Last updated May 26, 2026 08:42 UTC
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Sound waves shed light on nerve damage in rare disease
Knowledge-focused Recruiting nowThis study uses high-resolution ultrasound to look at nerves in people with Friedreich ataxia, a rare genetic disease that damages the nervous system and heart. Researchers want to see if nerve size and blood flow can help track how the disease progresses. The study involves 14 a…
Matched conditions: FRIEDREICH ATAXIA
Sponsor: Centre Hospitalier Universitaire de Nice • Aim: Knowledge-focused
Last updated May 26, 2026 08:40 UTC
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Researchers launch 5-Year safety check for Friedreich's ataxia drug
Knowledge-focused Recruiting nowThis study follows 300 people with Friedreich's ataxia who are taking or starting omaveloxolone (SKYCLARYS®). Researchers will collect safety information over up to 5 years, focusing on serious side effects and heart or liver problems. Participants continue their normal care, and…
Matched conditions: FRIEDREICH ATAXIA
Sponsor: Biogen • Aim: Knowledge-focused
Last updated May 26, 2026 08:35 UTC
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New study tracks heart disease in friedreich ataxia patients
Knowledge-focused Recruiting nowThis study aims to learn more about how heart disease develops and changes over time in people with Friedreich ataxia, a rare genetic disorder. Researchers will monitor 65 participants aged 6 and older using heart imaging to track key measures like heart muscle thickness and mass…
Matched conditions: FRIEDREICH ATAXIA
Sponsor: Lexeo Therapeutics • Aim: Knowledge-focused
Last updated May 22, 2026 13:52 UTC