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Administering small molecules that mimic factors naturally found in the brain to a mouse model of Friedreich’s ataxia increased levels of frataxin expression in both the brain and heart — two organs highly affected in people with the disease. The study, “Neurotrophic factor and cytokine mimetics as new potential therapeutic agents for Friedreich’s…

New viral vectors were found to effectively transfer a normal and protein-producing frataxin gene into major tissues affected by Friedreich’s ataxia — including the brain and the heart — in non-human primates, a new study showed. The study was presented by Holger Patzke of Voyager Therapeutics at IARC 2017 in a presentation Saturday titled “Intravenous delivery of AAV gene therapy to…

Using a gene therapy strategy similar to one previously shown to reverse heart problems in a Friedreich’s ataxia mouse model, researchers also corrected sensory symptoms associated with the disease in another mouse model. These results suggest that gene therapy may effectively treat different features of Friedreich’s ataxia. Hélène Puccio with the Institut de Genetique…

Omaveloxolone (RTA 408), an investigation medicine by Reata Pharmaceuticals, improves neurological function in people with Friedreich’s ataxia, according to results from part one of the MOXIe trial presented today at IARC 2017. David Lynch, a pediatric neurologist at Children’s Hospital of Philadelphia and a principal investigator for the Phase 2 clinical study, delivered these…

Giving mice who serve as a model of cardiac defects in Friedreich’s ataxia a molecule known to improve heart function in cardiac mice did help to improve how their hearts worked — but only if a mitochondrial protein, known as SIRT3, was present, a study reported at IARC 2017. Angel Martin, of…

Patients with spinocerebellar ataxia 38 (SCA38) showed improved scores in two different scales — SARA (Scale for the Assessment and Rating of Ataxia) and ICARS (International Cooperative Ataxia Rating Scale) — after about 24 weeks of using docosahexaenoic acid (DHA) as a supplement, a study reported at IARC 2017. DHA is…

Many scientists working on ataxia therapies focus on the movement, balance and heart problems that are the hallmarks of these neurological diseases. But a panel of five patients and caregivers at IARC 2017 asked researchers Friday not to forget the vision, hearing, and speech difficulties that a lot of patients…

Ataxia patients who took part in a new, at-home program for speech impairment showed significant improvements in their ability both to speak more clearly and retain vocal control, results of a small four-week study showed. These findings were presented in an IARC 2017 poster session by Adam P. Vogel with the…

A new set of small molecules specifically targeting the ubiquitin degradation system inside cells was found to effectively increase frataxin protein levels in several cell types taken from Friedreich’s ataxia (FA) patients, and merits further investigation. The study reporting these findings, “Highly specific ubiquitin-competing molecules promote frataxin accumulation in Friedreich ataxia iPSC-derived neuronal…

Dr. Colin Meyer, the chief medical officer and vice president of Reata Pharmaceuticals, spoke Friday in a taped interview with reporter Patricia Inacio about omaveloxolone, an oral therapy in line to possibly become the first FDA-approved treatment for Friedreich’s ataxia. A Phase 2 clinical trial, called MOXIe (NCT02255435), is moving into a second and…