News

FA likely marked by problems making mitochondrial proteins

A deficiency of frataxin, the protein whose lack causes Friedreich’s ataxia, leads to problems with mitochondrial translation — the molecular process by which new proteins are made in the mitochondria, the so-called powerhouse of the cell responsible for energy production — according to a new study. The findings specifically…

Voyager selects gene therapy candidate for future clinical trials

Voyager Therapeutics has selected a lead gene therapy candidate to advance into clinical trials with the aim of correcting the genetic defect that causes Friedreich’s ataxia (FA). With this decision, which was made with Neurocrine Biosciences, the companies expect to begin clinical trials in 2025. “The nomination…

FA community voices eagerness, expectations for gene therapy

People in the Friedreich’s ataxia (FA) community await the possibility of gene therapies for their disease, with many saying they would participate in gene therapy trials regardless of the potential for side effects or other consequences. Around half of the survey’s 137 respondents expected that such treatment — delivering…

Energy production complex in mitochondria may be FA target

A deficiency in the frataxin protein, which is the underlying cause of Friedreich’s ataxia (FA), alters a process in a large protein structure in mitochondria critical for energy production, a cell-based study suggests. Specifically, this deficiency affects the formation of iron-sulfur clusters — which are specialized molecules needed for…