News

FA Mouse Models May Not Fully Mimic Human Disease

Truncated forms of frataxin, the protein that is lost in people with Friedreich’s ataxia (FA), were found predominantly in mouse tissues, contrasting with the dominance of non-truncated frataxin in human tissue, a study found. The findings have implications for mouse models used to mimic the condition in disease research…

FDA Puts Hold on CTI-1601 Clinical Development

The U.S. Food and Drug Administration has put a hold on the clinical development of CTI-1601, an investigational treatment for Friedreich’s ataxia (FA) that is being developed by Larimar Therapeutics. According to a press release from Larimar, the hold was put in place after Larimar notified the…

Supporters Set to ‘Flex Their Muscles’ for Awareness Month in May

Events are underway globally to mark Friedreich’s Ataxia Awareness Month, observed each May, to heighten awareness about the neuromuscular disease and those it affects. Awareness Day is May 15. Patients, caregivers, and advocates worldwide will flex their muscles, sport temporary tattoos, and participate in research presentations — all in the…

Healx Joins With FARA, Ataxia UK for Work on Potential FA Therapies

Healx has partnered with the Friedreich’s Ataxia Research Alliance (FARA) and Ataxia UK to combine its drug discovery expertise with the organizations’ patient and scientific insights in order to develop potential treatments for Friedreich’s ataxia. “We believe that partnership and collaboration — sharing knowledge and resources —…