Autosomal recessive degenerative and progressive cerebellar ataxia

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 phase 2 trial tests a powerful mix of short-course radiation, chemotherapy (mFOLFOX6), an immunotherapy (PD-1 antibody), and a targeted drug (cetuximab or bevacizumab, depending on genetic markers) in people with high-risk rectal cancer that is mismatch repair proficient (pM…

This trial tests a personalized treatment plan for people with advanced colorectal cancer that has spread to the liver and stopped responding to standard treatments. The approach combines a liver-directed chemotherapy (HAIC), a targeted therapy (fruquintinib or cetuximab), and an…

This study tested a malaria drug called artesunate in 20 people with Friedreich ataxia, a rare genetic disorder that affects movement and coordination. The goal was to find a safe dose that could help control iron levels in cells, which may slow the disease. Researchers started w…

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

This study looked at whether taking an NAD+ precursor (a vitamin-like supplement) along with exercise training can improve aerobic capacity (how well the body uses oxygen during exercise) in people with Friedreich's ataxia. The trial included 75 participants aged 10 to 40. Resear…

This pilot study tested the drug elamipretide in 20 people with Friedreich ataxia to see if it could safely improve vision loss. Participants received either a low or high dose, and researchers measured changes in eyesight. The study is complete, but results are not yet available…

This study tested whether 12 weeks of respiratory strength training could improve breathing and swallowing in a person with Friedreich's ataxia. The participant did breathing exercises against resistance and had tests before and after the training. Because only one person took pa…

This completed study from the National Institute of Neurological Disorders and Stroke aimed to better understand how the brain controls movement and what goes wrong in movement disorders like Parkinson's disease, Tourette's syndrome, and dystonia. Over 1,200 adults—both patients …

This study follows 40 people with Friedreich's ataxia, a hereditary neurological condition that affects movement and speech, to see how their oral communication changes over one year. Researchers will assess speech clarity, voice quality, and hearing using speech therapy evaluati…

This study looked at 120 people with Friedreich's ataxia, a rare genetic disease that causes progressive movement problems. Researchers examined tiny interruptions in the DNA expansion that causes the disease to see if they influence when symptoms start and how severe they become…

This pilot study explored whether people with Friedreich's ataxia (FA) have problems making certain hormones, like cortisol and testosterone. Researchers measured hormone levels in blood samples from 11 FA patients and 15 healthy volunteers. The goal was to better understand how …

This study looked at 10 adults with Friedreich's ataxia to find a way to measure frataxin mRNA in blood and spinal fluid. The goal was to create a tool that can tell if treatments are working to increase frataxin levels in the brain. Researchers used tiny particles called exosome…

This study tested a wrist-worn device called the Personal KinetiGraph (PKG) to see if it can accurately measure movement problems in people with Parkinson's disease. Nineteen participants wore the device while researchers compared its readings to standard clinical tests and video…

This study followed 1200 people with Friedreich's ataxia over time to learn how the disease progresses. Researchers collected health exams and lab results to create a natural history of the condition. The goal was to develop better tools for future clinical trials and improve pat…