Marta Figueiredo, PhD,  science writer—

Etravirine — an approved anti-viral therapy for HIV — is expected to soon enter clinical testing as a potential new treatment for Friedreich’s ataxia (FA) in a pilot Phase 2 trial taking place in Italy. The proof-of-concept study (NCT04273165), called FAEST1, which is being conducted…

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…

Removal of the genetic defect that causes Friedreich’s ataxia (FA) in heart cells derived from a patient with heart disease associated with FA reversed the cells’ abnormal molecular profile, a study shows. This gene-editing strategy could be a useful tool for regenerative medicine aimed at treating heart defects associated…

Muscle stimulation through focal vibrations produced by a specialized device may prevent further deterioration of motor function in children with cerebellar ataxia, but not in Friedreich’s ataxia patients, according to a small study. The study, “Non-invasive Focal Mechanical Vibrations Delivered by Wearable Devices: An Open-Label Pilot Study…

A treatment approach that uses genetically engineered proteins was able to significantly increase the levels of expression of the FXN gene, which is mutated in Friedreich’s ataxia, allowing for greater frataxin protein to be produced in patients’ cells and in a mouse model of the disease. These findings suggest…

Friedreich’s ataxia patients produce less sweat than healthy people, which may be a useful biomarker of disease severity and progression, a recent study suggests. The study, “Sudomotor dysfunction is frequent and correlates with disability in Friedreich ataxia,” was published in the journal Clinical Neurophysiology. Patients…

Omaveloxolone, Reata Pharmaceuticals’ investigational candidate for the treatment of Friedreich’s ataxia (FA), has been granted orphan drug status from the European Medicines Agency. The EMA’s decision follows orphan drug designation from the U.S. Food and Drug Administration granted in June 2017. These designations are expected to…

The clinical benefits of stem cell therapy in Friedreich’s ataxia patients will be assessed in a small pilot study at the University of Bristol in the United Kingdom. Friedreich’s ataxia (FA) is a rare neurodegenerative disorder caused by mutations in the FXN gene, which contains instructions to produce frataxin, an essential…