News

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,…

Australian researchers have successfully turned stem cells from Friedreich’s ataxia (FA) patients into heart cells to study molecular anomalies that may contribute to this disease. These “heart cells in a dish” provide valuable information for the design of novel treatments. Their study, “Friedreich’s ataxia induced pluripotent stem cell-derived cardiomyocytes display electrophysiological…

An abnormal increase in protein acetylation leads to heart disease in Friedreich’s ataxia (FA) patients and may represent a new therapeutic target for early intervention, a study finds. The study “Progressive mitochondrial protein lysine acetylation and heart failure in a model of Friedreich’s ataxia cardiomyopathy,” appeared in Plos One. The authors found a close…

Friedreich’s ataxia (FRDA) patients have less mitochondria than other people because of the mutation in the frataxin (FXN) gene that is associated with the disorder, according to two studies. University of California at Davis researchers also said that a multiple sclerosis therapy the United States and Europe have already approved…

Researchers detected alterations in cerebral brain functions of patients with Friedreich’s ataxia and believe they may mitigate the impact of motor impairments of the disease. The study, “Cerebral compensation during motor function in Friedreich ataxia: The IMAGE-FRDA study,” was published in the journal Movements Disorders. Friedreich’s ataxia (FA), the most common inherited ataxia is characterized by motor…