In Friedreich’s ataxia, insufficient frataxin protein in cells impairs the ability of mitochondria to control the production of harmful reactive oxygen species, which accumulates and ultimately triggers nerve cell death, research into a mouse model of the disease revealed. The study, detailing how harm done to mitochondria in nerve cells helps to promote…
Scientists at University College London have, for the first time, described the full sequence of faulty mitochondrial processes in a Friedreich’s ataxia mouse model. Findings show that dysfunctional mitochondria directly contribute to cell death through lipid peroxidation in disease-affected neurons – a degradation of cellular lipids that if prevented,…
Retrotope announced the completion of a first dosing cohort, and the opening of patient enrollment for a highest dose cohort, in its ongoing, 28-day clinical study of orally dosed RT001 in Friedreich’s ataxia (FA) patients. The treatment to date has been well-tolerated, with no serious adverse effects or dose limiting toxicities, the pharmaceutical company reported. “We…
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