Corticosteroid therapy is a potential therapeutic treatment for patients with Friedreich ataxia (FRDA) and nephrotic syndrome, according to a study titled “Friedreich Ataxia and nephrotic syndrome: a series of two patients,” published in the journal BMC Neurology. FRDA is a genetic, progressive, neurodegenerative disorder that causes…
In a new study entitled “Efficient attenuation of Friedreich’s ataxia (FRDA) cardiomyopathy by modulation of iron homeostasis-human induced pluripotent stem cell (hiPSC) as a drug screening platform for FRDA,” researchers suggest that the chemical compound deferiprone, a mitochondrial permeable iron chelator, is an effective drug to treat Friedreich’s ataxia-mediated cardiomyopathy by…
In a recent study entitled “FXN Promoter Silencing in the Humanized Mouse Model of Friedreich Ataxia” a team of researchers reported that the mechanisms leading to silencing of the FXN gene, the underlying cause of Friedreich ataxia (FA), is not confined to tissues and cells reported to be affected…
In a recent study entitled “Genome-Engineering Tools to Establish Accurate Reporter Cell Lines That Enable Identification of Therapeutic Strategies to Treat Friedreich’s Ataxia” a team of researchers described a new system that allows the identification of novel molecular pathways responsible for Friedreich’s Ataxia, as well as potential new…
In a recent study entitled “Biological and clinical characteristics of the European Friedreich’s Ataxia Consortium for Translational Studies (EFACTS) cohort: a cross-sectional analysis of baseline data,” researchers describe the biological and clinical characteristics of patients with Friedreich’s Ataxia as a model of clinical outcomes to be…
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