Adolescents with Friedreich’s ataxia (FA) are now being dosed with nomlabofusp as part of a placebo-controlled Phase 1 clinical study that’s testing Larimar Therapeutics‘ subcutaneous, or under-the-skin, injection therapy. Data from the Phase 1 study, and from other clinical testing of nomlabofusp, are expected by the middle…
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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…
Throughout 2024, Friedreich’s Ataxia News brought readers detailed coverage of the latest clinical research related to Friedreich’s ataxia (FA), as well as updates on scientific breakthroughs and developments in treatment. Here are the top 10 most-read stories of last year, each with a brief description. We’re excited to continue…
Note: This story was updated Dec. 19, 2024, to clarify that the BOLD study does include measures of efficacy. Since its 2023 launch as the first and — to date — only approved treatment for Friedreich’s ataxia in the U.S., Skyclarys (omaveloxolone) has benefited most patients by either…
A genetic analysis of connective tissue cells from a woman with late-onset Friedreich’s ataxia (FA) and her younger sister, who did not have the disease, found differences in the activity levels of almost 400 genes in the affected sister, according to a recent study. Genes with the most altered…
Data from wearable sensors that are designed to collect real-world information about a person’s ability to function in daily life correlated well with standard clinical assessments of disease severity among people with Friedreich’s ataxia (FA), a study finds. These types of remote monitoring tools could help to better track…
A study of heart health with vatiquinone, an investigational oral Friedreich’s ataxia (FA) therapy, found no increased risk of cardiac problems in healthy adults, including when doses about 3.5 times above the therapeutic dose were evaluated, scientists reported. Blood levels of vatiquinone did not correlate with changes in…
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…
Key takeaways from the webinar include the complexity of the relationship between FA patients and caregivers, and the importance of practicing open communication and empathy and asking for help when necessary.