News

On a hot, Texas afternoon, Madelyn Raynsford’s home buzzes with the practical chaos of preparing for a new life: half-packed boxes, baby gear spread out, her two dogs lounging nearby. Her computer screen is filled with tabs researching safe and adaptive feeding options. Baby bottles top the list — not…

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…

People with Friedreich’s ataxia (FA) show distinct changes in the blood proteins neurofilament light chain (NfL) and tau that could help track the disease and measure the effects of treatment, a study finds. In younger patients, NfL levels were sharply higher than in healthy people, making it a promising…

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…

Genetic mutations causing Friedreich’s ataxia (FA) and influencing disease severity may be more complex than previously understood, according to research from the University of Oklahoma. Sanjay Bidichandani, PhD, genetics section chief at the university’s medical college, used a new genetic sequencing technique to find “spelling errors” in the FXN gene…

An experimental therapy may help increase the protein that’s missing or deficient in Friedreich’s ataxia (FA) in people with a rare mutation in the FXN gene, a new study suggests. The therapy targets a rare mutation, known as c.165+5G>C, which has been reported in only three patients to date. The…

The U.S. Food and Drug Administration (FDA) has granted breakthrough therapy designation to LX2006, a gene therapy for cardiomyopathy in Friedreich’s Ataxia (FA). The status means the Lexeo Therapeutics candidate may be eligible for fast track review and extra FDA guidance during the regulatory process, which could…

A newly awarded $300,000 research grant will fund a study on heart fibrosis, or the accumulation of scar tissue, in people with Friedreich’s ataxia (FA). The Muscular Dystrophy Association (MDA) and Friedreich’s Ataxia Research Alliance (FARA) awarded the grant to researchers at Weill Cornell Medical Center and Indiana…

The U.S. Food and Drug Administration (FDA) has provided Larimar Therapeutics with clear expectations for an application seeking accelerated approval of nomlabofusp, its potentially disease-modifying therapy for Friedreich’s ataxia (FA), according to the drug developer. Accelerated approval is a type of conditional approval based on early clinical…