FRIEDREICH ATAXIA

This study tests a drug called omaveloxolone (SKYCLARYS) in children aged 2 to 15 with Friedreich's ataxia, a rare nerve disease that affects movement and balance. The drug is already approved for people 16 and older. In the first year, some children get the drug and some get a p…

This study is testing the long-term safety and effects of a daily injection called CTI-1601 in people with Friedreich's ataxia, a rare genetic disorder that damages the nervous system. About 85 adults and teens who have or haven't taken the drug before will receive it for an exte…

This study tests an approved FA drug (omaveloxolone) in 35 children aged 2 to 15 to see how their bodies process it and whether it is safe. The drug is already available for patients 16 and older. Researchers will monitor side effects, growth, and heart health over up to 240 week…

This is an early-stage study testing the safety of a new gene therapy for the heart damage caused by Friedreich's ataxia. The therapy uses a modified virus to deliver a healthy gene directly to the heart. Researchers will enroll 25 participants to find a safe dose and look for ea…

This study looks at whether omaveloxolone, a drug recently approved in France, can ease swallowing difficulties in people with Friedreich's ataxia. About 40 patients will fill out a swallowing questionnaire before starting the drug and again after six months. The goal is to see i…

This study aims to learn more about the daily challenges, quality of life, and costs for people with Friedreich Ataxia (FA). About 200 participants from Germany, Austria, and France will use a mobile app to report their experiences over six months. The goal is to better understan…

This 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…

This study watches how heart disease develops and changes over time in people with Friedreich ataxia, a rare genetic condition. Researchers will measure heart muscle thickness and mass using imaging. The goal is to learn more about the disease to help design future treatments. Ab…

This observational study follows 300 people with Friedreich's ataxia who are taking the prescribed drug omaveloxolone (SKYCLARYS®). Researchers will collect safety information from routine doctor visits over up to 5 years. The goal is to monitor serious side effects, especially h…