FRIEDREICH ATAXIA

This study looks at the long-term safety of the drug vatiquinone in 130 people with Friedreich ataxia who have already taken it in earlier studies. Researchers will track side effects and measure how the disease progresses over three years using a standard rating scale. The goal …

This study tests an approved drug for Friedreich's ataxia (called omaveloxolone or SKYCLARYS) in children aged 2 to 15. The goal is to see how the drug moves through their bodies and whether it is safe. About 33 children will take the drug in two parts, first a single dose and th…

This early-stage study tests a one-time gene therapy called LX2006 in 8 people with Friedreich's ataxia who also have heart muscle disease. The therapy delivers a working copy of the frataxin gene directly to heart cells to improve heart function. Researchers will monitor safety …

This 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…

This study aims to create a simple test to determine the risk of falling for people with neuromuscular disorders like muscular dystrophy, ALS, and myasthenia gravis. Researchers will evaluate how well a series of physical tasks (like standing up, walking, and balancing) can predi…