FRIEDREICH ATAXIA
Clinical trials for FRIEDREICH ATAXIA explained in plain language.
Never miss a new study
Get alerted when new FRIEDREICH ATAXIA trials appear
Sign up with your email to follow new studies for FRIEDREICH ATAXIA, keep track of the ones that matter, and come back to a personal dashboard instead of checking manually.
By submitting, you agree to our Terms of use
-
Gene therapy targets heart damage in rare neurological disease
Disease control OngoingThis early-stage study tests a gene therapy called LX2006 in 8 people with Friedreich's ataxia who also have heart muscle disease. The treatment delivers a working copy of the frataxin gene to heart cells through a single IV infusion. Researchers will monitor safety and heart fun…
Matched conditions: FRIEDREICH ATAXIA
Phase: PHASE1, PHASE2 • Sponsor: Lexeo Therapeutics • Aim: Disease control
Last updated May 17, 2026 07:06 UTC
-
Gene therapy for rare heart condition put on hold before it began
Disease control TerminatedThis study was designed to test a new gene therapy called ASP2016 for heart problems in people with Friedreich Ataxia, a rare genetic disease. The goal was to check safety and how well people tolerated the treatment, which involved a single infusion plus steroids to protect the t…
Matched conditions: FRIEDREICH ATAXIA
Phase: PHASE1 • Sponsor: Astellas Gene Therapies • Aim: Disease control
Last updated May 17, 2026 06:48 UTC
-
Hope for friedreich ataxia: Long-Term vatiquinone safety trial underway
Disease control OngoingThis study looks at the long-term safety of a drug called vatiquinone in people with Friedreich ataxia who have already taken it in earlier studies. About 130 participants will be followed for up to 3 years to monitor side effects and track changes in their movement and coordinat…
Matched conditions: FRIEDREICH ATAXIA
Phase: PHASE3 • Sponsor: PTC Therapeutics • Aim: Disease control
Last updated May 13, 2026 16:01 UTC
-
New test could predict falls in muscle disease patients
Knowledge-focused ENROLLING_BY_INVITATIONThis study aims to create a simple test to predict fall risk in people with neuromuscular disorders like muscular dystrophy, ALS, and myasthenia gravis. Researchers will measure how well participants perform tasks like standing up from a chair, walking, and balancing. The goal is…
Matched conditions: FRIEDREICH ATAXIA
Sponsor: LMU Klinikum • Aim: Knowledge-focused
Last updated May 07, 2026 18:39 UTC