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Friedreich ataxia (FRDA) is a progressive, inherited, neurodegenerative disease for which there is currently no cure or approved treatment. FRDA is caused by deficits in the production and expression of frataxin, a protein found in the mitochondria that is most likely responsible for regulating iron-sulfur cluster enzymes within the cell. A decrease in frataxin causes dysfunction of adenosine triphosphate synthesis, accumulation of mitochondrial iron, and other events leading to downstream cellular dysfunction. Areas covered: Therapeutic development for FRDA currently focuses on improving mitochondrial function and finding ways to increase frataxin expression. Additionally, the authors will review potential approaches aimed at iron modulation and genetic modulation. Finally, gene therapy is progressing rapidly and is being explored as a treatment for FRDA. Expert commentary: The collection of multiple therapeutic approaches provides many possible ways to treat FRDA. Although the mitochondrial approaches are not thought to be curative, as the primary frataxin deficit will remain, they may still produce improvements in quality of life and slowing of progression. Therapies aimed at frataxin restoration are more likely to truly modify the disease, with gene therapy as the best possibility to alter the course of the disease from both a cardiac and neurological perspective.

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Objective In vitro, in vivo, and open‐label studies suggest that interferon gamma (IFN‐γ 1b) may improve clinical features in Friedreich Ataxia through an increase in frataxin levels. The present study evaluates the efficacy and safety of IFN‐γ 1b in the treatment of Friedreich Ataxia through a double‐blind, multicenter, placebo‐controlled trial. Methods Ninety‐two subjects with FRDA between 10 and 25 years of age were enrolled. Subjects received either IFN‐γ 1b or placebo for 6 months. The primary outcome measure was the modified Friedreich Ataxia Rating Scale (mFARS). Results No difference was noted between the groups after 6 months of treatment in the mFARS or secondary outcome measures. No change was noted in buccal cell or whole blood frataxin levels. However, during an open‐label extension period, subjects had a more stable course than expected based on natural history data. Conclusions This study provides no direct evidence for a beneficial effect of IFN‐γ1b in FRDA. The modest stabilization compared to natural history data leaves open the possibility that longer studies may demonstrate benefit.

The growth in professional development for the infant‐early childhood workforce has necessitated the implementation of novel, sustainable approaches to meet infant early childhood mental health (IECMH) training and reflective supervision consultation (RSC) needs. The 12‐month pilot of a US statewide reflective consultation (RC) group model included IECMH consultants, grant specialists, supervisors, and program managers (n = 38) and their group reflective consultants (n = 6). The pilot evaluation provided an opportunity to design a study that assessed the impact of RC on infant‐early childhood professionals. The mixed‐methods study included an assessment of consultees’ reflective practice skills and experience of their work. Findings included consultees’ self‐reported increased reflective practice self‐efficacy and increased use of reflective practice skills. While there were no changes in the Maslach Burnout Inventory (MBI) personal accomplishment, emotional exhaustion, or depersonalization results, qualitative findings indicated a decrease in burnout and an increase in relationship‐based practice across professional roles. This unique pilot provides an example of an organizational approach to instituting RC with a broad spectrum of infant‐early childhood professionals and yields valuable information about the impacts of RC models on such professionals’ work experience and professional practice.