Reata Pharmaceuticals is planning to ask the U.S. Food and Drug Administration (FDA) to approve omaveloxolone to treat people with Friedreich’s ataxia (FA) early in 2022, following the completion of a preliminary meeting with the agency. “Omaveloxolone could be the first drug approved for the treatment of Friedreich’s…
Researchers have developed a new system that might better measure walking or gait problems in people with Friedreich’s ataxia and other nerve and muscle conditions. The study describing this method was published in the journal Sensors, as “Gauging Gait Disorders with a Method Inspired by Motor Control…
Eurordis opened a campaign, called Rare 2030 Action, that is seeking to establish a European action plan for rare diseases to ensure that none of the 30 million people in Europe living with rare diseases are left behind by the start of a new decade. As part of…
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…
Leriglitazone, an oral treatment candidate for Friedreich’s ataxia (FA), can increase frataxin levels and reduce neuronal degeneration in cell and animal models of the disease, a study found. Along with promising clinical findings from a Phase 2 clinical trial (NCT03917225), the results support the use of leriglitazone, and the…
The National Organization for Rare Disorders (NORD)’s RareLaunch training program will host two days of free virtual workshops in December, with the aim of empowering leaders to start non-profit organizations and research programs to help people with rare diseases. “The RareLaunch program is…
The Friedreich’s Ataxia Research Alliance (FARA), the CureFA Foundation, and fara Australia are supporting four research projects that aim to better understand the biology underlying Friedreich’s ataxia (FA) and pave the way for future treatment approaches, FARA announced in a press release. The Award for…
A majority of rare disease patients using telehealth during the COVID-19 pandemic thought the experience positive, and many would like the option of continuing its use in future appointments, a series of surveys found. The surveys were conducted by the National Organization for Rare Disorders (NORD) and involved more than 800…
IXICO has entered a five-year collaboration with the Friedreich’s Ataxia Research Alliance (FARA) and joined the TRACK-FA Neuroimaging Consortium, a group of academic and industry partners that are conducting a natural history study focused on tracking brain and spinal cord changes in patients with Friedreich’s ataxia (FA).
Measuring blood levels of two proteins, N-terminal pro-brain natriuretic peptide (NT-proBNP) and troponin, may provide useful information about heart health in people with Friedreich’s ataxia (FA), a study suggests. The study, “Significance of NT-proBNP and High-Sensitivity Troponin in Friedreich Ataxia,” was published in the …
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