News

The U.S. Food and Drug Administration (FDA) has granted orphan drug status to Jotrol, a potential new therapy for Friedreich’s ataxia (FA), says the medication’s developer, Jupiter Orphan Therapeutics. The FDA decision “positions our Jotrol product as a potential treatment for FA … patients around the world,” Jupiter CEO…

Research into how abnormalities in the large area of the brain are part of Friedreich’s Ataxia disease processes will advance understanding and improve the chances of finding new treatments, researchers from Australia’s Monash University contend in a review. But research needs to focus on larger, multi-modal studies that will allow both…

The first International Ataxia Research Conference, in 2015, was a milestone in the quest to find cures for the world’s 40 ataxias, diseases whose hallmark is the loss of muscular coordination. Held in Windsor, England, it was the largest global convention yet to bring together patients, doctors, patient advocacy groups, researchers, pharmaceutical companies, regulators…

Researchers at the Children’s Hospital of Philadelphia (CHOP) have linked the presence of diabetes in Friedreich’s ataxia (FA) patients to more severe symptoms and a reduced capacity to manage daily living tasks. These insights suggest that identifying patients with diabetes and more actively managing their disease might help improve their health…

Chondrial Therapeutics‘ CTI-1601, an investigative therapy for Friedreich’s ataxia (FA), has been granted orphan drug status by the U.S. Food and Drug Administration (FDA). “We are very pleased to receive orphan drug designation for CTI-1601. Our team is working to complete all Investigational New Drug (IND) enabling studies so that…

The U.S. Food and Drug Administration (FDA) agreed that Reata Pharmaceuticals can use the modified Friedreich’s Ataxia Rating Scale (mFARS) as a primary endpoint for the second part of its possibly pivotal Phase 2 clinical trial assessing omaveloxolone in treating Friedreich’s ataxia, the company announced. This decision followed Reata’s request that the FDA confirm its earlier guidance that,…

Reata Pharmaceuticals sold some of its shares, generating sales of approximately $115.9 million to be used to support the clinical development of the company’s pipeline, including omaveloxolone, its lead investigative product for Friedreich’s ataxia, according to a press release. Friedreich’s ataxia is caused by a genetic defect…

Two small molecules were shown to increase the mRNA and protein levels of frataxin (FXN) in animal models of Friedreich’s ataxia. According to a report published at Neuropharmacology, these compounds could have therapeutic potential to treat FXN deficits in Friedreich’s ataxia (FA). Friedreich’s ataxia is caused by reduced levels of the mitochondrial…

A protein called SIRT3 was essential to restore the heart contractility and energy metabolism that can result from an energy-promoting supplement called nicotinamide mononucleotide (NMN), researchers report from a study in Friedreich’s ataxia mouse models. The study, “Nicotinamide mononucleotide requires SIRT3 to improve cardiac function and bioenergetics in a Friedreich’s ataxia cardiomyopathy model,” was…

Seven microRNAs that circulate in the blood may serve as biomarkers that help clinicians to diagnose Friedreich’s ataxia, according to results of a recent study. High levels of one of these molecules, called miR-323-3p, also has the potential to identify those patients with disease-associated cardiomyopathy (heart failure), the researchers report. Their study,…