Ana Pena, PhD,  —

Patients with Friedreich’s ataxia (FRDA) who have trouble standing upright are at greater risk of losing their ability to walk, a report based on a large natural history study found. Standing and balance tests could be used to separate groups of…

Retrotope has dosed the first patient with RT001, an experimental oral treatment for Friedreich’s ataxia (FA), in a Phase 2/3 clinical trial currently recruiting adult patients at sites in the U.S. Three clinical centers are now open for enrollment — Collaborative Neuroscience Network in California,…

Speech disorders like dysarthria are a common and disabling symptom of Friedreich’s ataxia (FA), but they are largely ignored in clinical trials. More research is needed to develop better ways of assessing and intervening to address them, a review study reports. It also suggests various assessment tests and treatment approaches…

Scoliosis, an abnormal curvature of the spine, is highly prevalent in children with Friedreich’s ataxia (FA), predominantly caused by right-sided thoracic curves and commonly associated with a “hunchback,” a study describes. Early onset of scoliotic deformities, before age 10 and when bones are still immature, also is frequent…

Leriglitazone (MIN-102), an investigational oral treatment by Minoryx Therapeutics, has been granted orphan drug status by the U.S. Food and Drug Administration (FDA) for treating patients with Friedreich’s ataxia (FA). Orphan drug status qualifies Minoryx for various incentives meant to expedite clinical testing and possible approval…

MOXIe, the Phase 2 study of oral omaveloxolone (omav), is now the first trial to show significant neurological benefits in a broad population of patients with Friedreich’s ataxia (FA), Reata Pharmaceuticals announced last week. Its major finding was a…

MOXIe, the Phase 2 study of oral omaveloxolone that may prove pivotal to it becoming the first approved targeted treatment for Friedreich’s ataxia (FA), succeeded in showing significant benefits to patients because of years of “groundwork” laid down by a patient group and researchers at Reata Pharmaceuticals,…

Topline results of Reata‘s MOXIe trial show that almost one year of treatment with the investigational oral therapy omaveloxolone (RTA 408) is well-tolerated and leads to significant benefits in neurological function in patients with Friedreich’s ataxia (FA), meeting the Phase 2 trial’s primary goal.

Friedreich’s ataxia caused by GAA repeat expansion and point mutations — a rare origin of the disease — is present in the Brazilian population and should be considered for diagnostic testing and genetic counseling in the country, a study shows. The study, “Frequency and Genetic Profile of Compound…

Treatment with methylprednisolone, an anti-inflammatory medication, had no evident benefit in children and adults with Friedreich’s ataxia (FA) enrolled in a pilot clinical trial conducted at the Children’s Hospital of Philadelphia (CHOP). Oral therapy with methylprednisolone failed to improve patients’ overall mobility and neurological function, although three children…

A naturally occurring variant of ISCU2 protein can have the same molecular role as frataxin, an experimental study has found. This discovery may pave the way for the development of new strategies to bypass the effects of frataxin deficiency, which is known to cause …

People with Friedreich’s ataxia (FRDA) have fewer small nerve fibers in their skin compared with healthy subjects, and the greater a patient’s defects in the frataxin (FXN) gene, the more the nerve fiber loss, a…

A discovery made in plants may shed light on the genetic mechanism underlying Friedreich’s ataxia (FA) and open the door to new ways to treat the disease, a study reports. Looking at genetic alterations in plants similar to the ones found in the…

People with Friedreich’s ataxia (FA) have alterations in the connectivity of different brain regions, which most likely is associated with the disease’s neurological symptoms. Researchers used magnetic resonance imaging (MRI) to analyze the brains of patients…