News

Leriglitazone, an oral treatment candidate for Friedreich’s ataxia (FA), is well-tolerated and can significantly prevent ataxia worsening and iron buildup in the brain, according to top-line data from a Phase 2 trial. The proof-of-concept study, called FRAMES (NCT03917225), also demonstrated that the therapy was able to improve a series…

Larimar Therapeutics completed dosing for its single-ascending dose (SAD) Phase 1 clinical trial of CTI-1601, a modified version of frataxin, in the treatment of Friedrich’s ataxia (FA), the company announced. A safety review committee cleared the trial to continue after evaluating preliminary blinded data from each of the…

PTC Therapeutics has launched a Phase 3 clinical trial to evaluate its investigational oral therapy vatiquinone (PTC743) in children and young adults with Frederich’s ataxia (FA). The international trial had experienced earlier delays due to the COVID-19 pandemic. Called MOVE-FA (NCT04577352), it is meant to…

To empower and equip members of the rare disease community to engage state leaders in matters of importance to patients and their families, the National Organization for Rare Disorders (NORD) has launched an initiative across the U.S. Its goal is to establish a Rare Disease Advisory Council (RDAC)…

The first wave of COVID-19 in Europe severely disrupted access to care and raised stress and anxiety in people with rare diseases, negatively affecting their health and well-being, according to a survey conducted by Eurordis-Rare Diseases Europe. “People living with rare diseases in Europe have found themselves caught as collateral…

After nearly one year of its use, omaveloxolone (RTA 408) significantly improved neurological function across several clinical measures in people with Friedreich’s ataxia (FA), and was generally safe and well tolerated, according to results from the MOXIe Phase 2 trial.  These findings were published…

Leaders in the U.S. rare disease community came together recently for a webinar to present helpful information on how to start a nonprofit and patient registry. They shared about how their respective organizations came to be, as well as the benefits of creating patient registries and how they can help…

The Friedreich’s Ataxia Research Alliance (FARA) has shared a list of three clinical studies and one global registry that are open and enrolling people with Friedreich’s ataxia (FA) in the search for more effective therapies. This rare disease is caused by abnormally low levels of a protein called…

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 gene therapy startup AavantiBio has launched with $107 million in funding to advance its lead clinical program for the treatment of Friedreich’s ataxia (FA). Early this year, the Boston-based company received a $1 million grant from the Muscular Dystrophy Association (MDA)’s Venture Philanthropy Fund…