Using a gene therapy strategy similar to one previously shown to reverse heart problems in a Friedreich’s ataxia mouse model, researchers also corrected…
Articles by Patricia Inácio, PhD
Omaveloxolone (RTA 408), an investigation medicine by Reata Pharmaceuticals, improves neurological function in people with Friedreich’s ataxia, according to results from part…
Giving mice who serve as a model of cardiac defects in Friedreich’s ataxia a molecule known to improve heart function…
Patients with spinocerebellar ataxia 38 (SCA38) showed improved scores in two different scales — SARA (Scale for the Assessment and Rating…
Ataxia patients who took part in a new, at-home program for speech impairment showed significant improvements in their ability both to…
A new set of small molecules specifically targeting the ubiquitin degradation system inside cells was found to effectively increase frataxin protein…
The degree of genetic defects in the frataxin gene appears to result in adults with Friedreich’s ataxia having a relatively smaller…
Synthetic molecules were able to increase frataxin protein levels in cells taken from Friedreich’s ataxia patients to levels near those…
Patients with different types of spinocerebellar ataxias showed significant improvement in their ability to hear and perceive complex sounds —…
Clinical trials to assess changes in the motor skills of Friedreich’s ataxia patients can and should include physical tests — specifically,…