FRIEDREICH ATAXIA

This study tested an experimental drug called vatiquinone in 146 people with Friedreich ataxia, a rare disease that affects movement and coordination. Participants took the drug or a placebo for 72 weeks to see if it slowed worsening of symptoms, measured by a standard rating sca…

This study tested the drug artesunate (usually used for malaria) in 20 people with Friedreich ataxia, a rare genetic disease that affects movement and coordination. The goal was to find a safe dose that might help control the disease by improving how the body handles iron. The tr…

This study tested a drug called RTA 408 (omaveloxolone) in 172 people with Friedreich's ataxia, a rare genetic disease that affects movement and coordination. The goal was to see if the drug is safe and can improve exercise ability and daily function. Participants took the drug o…

This study looked at whether a 12-week respiratory strength training program could improve swallowing and breathing in one person with Friedreich's ataxia. The participant had tests before and after training, including a camera look at swallowing, lung function measurements, and …

This study tested the safety and effectiveness of elamipretide for treating vision loss in people with Friedreich ataxia (FRDA). Twenty participants with advanced symptoms received either a low or high dose of the drug. The main goal was to see if it improved how many letters the…

This study aimed to find a way to measure frataxin mRNA in blood and spinal fluid from 10 adults with Friedreich's ataxia. Researchers used tiny particles called exosomes to detect the mRNA. If successful, this could become a tool to monitor treatments that aim to boost frataxin …