News

People with Friedreich’s ataxia should consult their cardiologist on a regular basis, as imaging of the heart considered with disease-related mutation repeats may identify those at high risk of cardiac problems up to five years before symptoms become evident, a study suggests. The study, “Predictors of Left Ventricular Dysfunction…

Rare disease-themed videos glowed on a large screen before an audience of people in wheelchairs, with crutches, and bearing oxygen tanks this Nov. 9 and 10 in San Francisco. Disorder: The Rare Disease Film Festival strives to eventually host a film about every one of the nearly 7,000 rare…

Minoryx Therapeutics’ lead investigational therapy leriglitazone (MIN-102) has been granted orphan drug designation by the European Commission for treating patients with Friedreich’s ataxia (FA). The European Commission’s decision follows a similar designation granted by the U.S. Food and Drug Administration in October. Orphan designation is given to investigative therapies…

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…

Reactivativation of the Nrf2 pathway, which is impaired in the nerve cells — or neurons — of patients with Friedreich’s ataxia (FA), leads to the re-establishment of proper neuron development and maturation, a mouse study shows. The study, “Nrf2 Induction Re-establishes a Proper Neuronal Differentiation Program in Friedreich’s…

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.

A Phase 2 trial testing leriglitazone as a potential oral treatment for Friedreich’s ataxia (FA) has recruited its target number of participants ahead of schedule. The multi-center, double-blind FRAMES study (NCT03917225) will assess leriglitazone in 39 participants, 12–60 years old, who were recruited in four and a…