FRIEDREICH ATAXIA
Clinical trials for FRIEDREICH ATAXIA explained in plain language.
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First-of-its-Kind gene therapy trial for rare disease heart damage
Disease control TerminatedThis was an early-stage study to test a new gene therapy called ASP2016 for heart problems in adults with Friedreich ataxia, a rare genetic disease. The main goals were to check if the treatment was safe and how well people could tolerate it. Participants would receive a single i…
Matched conditions: FRIEDREICH ATAXIA
Phase: PHASE1 • Sponsor: Astellas Gene Therapies • Aim: Disease control
Last updated Apr 02, 2026 02:11 UTC
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First gene therapy trial targets deadly heart damage in rare neurological disease
Disease control OngoingThis early-stage study is testing a new gene therapy, LX2006, for people with Friedreich's ataxia who have developed heart muscle damage. The therapy aims to deliver a healthy gene directly to heart cells to improve their function. Researchers will first check if the treatment is…
Matched conditions: FRIEDREICH ATAXIA
Phase: PHASE1, PHASE2 • Sponsor: Lexeo Therapeutics • Aim: Disease control
Last updated Mar 30, 2026 14:31 UTC
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Extended hope for rare neurological disease
Disease control OngoingThis study aims to see if the drug vatiquinone is safe and effective for people with Friedreich ataxia when taken for up to three years. It is for 130 participants who have already been taking vatiquinone in a previous trial. The main goal is to monitor for side effects while als…
Matched conditions: FRIEDREICH ATAXIA
Phase: PHASE3 • Sponsor: PTC Therapeutics • Aim: Disease control
Last updated Mar 30, 2026 14:29 UTC
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New test aims to stop dangerous falls for people with Muscle-Weakening diseases
Diagnosis ENROLLING_BY_INVITATIONThis study aims to create and validate a simple set of physical tests to better predict the risk of falling for people with various nerve and muscle disorders. Researchers will test 108 patients with conditions like muscular dystrophy and ALS using tasks like standing up from a c…
Matched conditions: FRIEDREICH ATAXIA
Sponsor: LMU Klinikum • Aim: Diagnosis
Last updated Mar 20, 2026 14:47 UTC