Marisa Wexler, MS,  senior science writer—

SGT-212, a gene therapy candidate for Friedreich’s ataxia (FA) that’s currently in early clinical testing, has been granted rare pediatric disease designation by the U.S. Food and Drug Administration (FDA). The FDA awards this status to experimental treatments that are designed to address rare diseases that primarily affect children,…

International Ataxia Awareness Day is coming up this Thursday, and organizations around the world are getting ready for this annual daylong opportunity to shine a light on what it’s really like to live with Friedreich’s ataxia (FA) and other ataxia types. “September 25, International Ataxia Awareness Day, is an…

Levels of mitochondrial complex 1 (MC1) — a group of proteins essential to the function of mitochondria, cells’ powerhouses — are lower than normal in the hearts of people with Friedreich’s ataxia (FA), a small study showed. The findings indicate that imaging of MC1 could be a useful biomarker…

The U.S. Food and Drug Administration (FDA) has declined to approve the oral therapy vatiquinone for treating children and adults with Friedreich’s ataxia (FA). In a complete response letter, the regulatory agency said it would not approve vatiquinone at this time because of a lack of adequate data…

A committee of Canada’s Drug Agency (CDA) has recommended that insurers should cover the cost of Skyclarys (omaveloxolone) for Friedreich’s ataxia (FA), but only under certain conditions, including a substantial reduction in the drug’s cost. “The CDA recommendation represents an important step toward changing that reality by…

Voyager Therapeutics expects to begin clinical testing of its experimental gene therapy for Friedreich’s ataxia (FA) in 2026. The company last year announced the selection of its FA gene therapy candidate, which is being developed in collaboration with Neurocrine Biosciences. At the time, Voyager had said…

Larimar Therapeutics plans to ask the U.S. Food and Drug Administration (FDA) this year to grant accelerated approval to nomlabofusp, its experimental treatment for Friedreich’s ataxia (FA). “The strong clinical and regulatory progress across our nomlabofusp program reinforces the timing of our planned Biologics License Application (BLA)…

Errors during repair when DNA gets damaged may give rise to the mutations that cause Friedreich’s ataxia, a study revealed. The study, “Recurrent DNA nicks drive massive expansions of (GAA)n repeats,” was published in PNAS. A cell’s DNA stores the instructions to make the proteins and…

The U.S. Food and Drug Administration (FDA) has given Solid Biosciences the go-ahead to start clinical testing of SGT-212, the company’s novel gene therapy candidate for Friedreich’s ataxia (FA). According to Solid, SGT-212 is the only experimental gene therapy of its kind to target “both neurologic and…

In adults with Friedreich’s ataxia (FA), treatment with nomlabofusp may increase levels of frataxin protein to at least half of what’s typically seen in people who don’t have the disease, according to new analysis of clinical trial data. The analysis also predicted that use of the experimental therapy…