Restoring normal frataxin production, the protein missing in Friedrich’s ataxia (FA), may become possible in the future, according to research recently published in the journal PLoS One. The study is titled “Disruption Of Higher Order DNA Structures In Friedreich’s Ataxia (GAA)n Repeats By PNA Or LNA Targeting,” and was…
Patients with late-onset Friedrich’s ataxia (LOFA) may present variable sensory nerve conduction, according to new research. The study, “Variable Sensory Nerve Conduction Parameters In Late Onset Friedreich Ataxia,” was published in the journal Muscle & Nerve. Nerve conduction studies are commonly performed by neurology specialists to assess the…
Researchers report that the analysis of the neurological changes occurring in patients with Friedrich’s Ataxia (FDRA) may serve as prediction factors of disease progression and be useful in the design of clinical trials to investigate future therapies. The findings suggest, namely, that clinical trials would benefit from recruitment of younger…
Recent research suggests that correcting the frataxin deficiency in neurons derived from Friedrich’s ataxia (FA) patients may not only stop disease progression, it may also lead to clinical improvement by rescuing dysfunctional surviving neurons. The study, “Friedreich ataxia induced pluripotent stem cell-derived neurons show a cellular phenotype that is corrected…
A new study from the University of California reported several unknown neurobehavioral deficits observed in an animal model for Friedrich’s ataxia (FA) that can be used to assess the effect of potential drug therapies. The study, “Neurobehavioral deficits in the KIKO mouse model of Friedreich’s ataxia,” was…
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