Forest Ray PhD,  —

Scientists on either side of the Atlantic have teamed up, through the new Oxford-Harrington Rare Disease Centre (OHC), to address unmet needs in rare disease research and deliver “major clinical impact” to patients. And now, the international collaboration between the University of Oxford, in England, and the Harrington Discovery…

The nonprofit EndFA has launched a free mobile phone and tablet app to help connect, empower, and better inform people with Friedreich’s ataxia (FA) wherever they live. With the mission to “Power the Cure!,” the FA App is designed to connect members of the FA community to each other…

A clinical trial will assess whether supplementation with calcitriol, the active form of vitamin D, can boost frataxin production in people with Friedreich’s ataxia (FA). The Biochemistry of Oxidative Stress Group (BOSG) at the Biomedical Research Institute of Lleida (IRBLleida) and the University of Lleida, in Spain, announced their…

The U.S. Food and Drug Administration (FDA) has given rare pediatric disease and orphan drug designations to LX2006, Lexeo Therapeutics‘ investigational gene therapy for people with Friedreich’s ataxia (FA). The designations each provide benefits meant to ease the therapy’s path through development. Rare pediatric disease status is provided to…

People with Friedreich’s ataxia (FA) are encouraged to enroll in three clinical trials evaluating the effects of dietary supplements on exercise, the safety and efficacy of the experimental therapy vatiquinone, and the utility of wearing sensors to monitor the disease’s progression and severity. The Friedreich’s Ataxia Research Alliance…

The U.S. Food and Drug Administration (FDA) has asked Reata Pharmaceuticals to request a preliminary meeting to discuss submitting a new drug application (NDA) for omaveloxolone, an investigational therapy for Friedreich’s ataxia (FA). Reata was due to hold a Type C meeting (unrelated…

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 —…

Stealth BioTherapeutics is planning a Phase 2a clinical trial of elamipretide (SS-31) as a treatment for people with vision loss and/or heart disease associated with Friedreich’s ataxia (FA). The company expects to use data from this study — planned to start later this year — in designing a pivotal…

Over about two weeks, more than 74,000 people worldwide have signed a petition from the Friedreich’s Ataxia Research Alliance (FARA) requesting that omaveloxolone (omav) be made available to people with Friedreich’s ataxia (FA) as quickly as possible. The petition specifically asks Reata Pharmaceuticals to submit a new drug…

The two COVID-19 vaccines that recently received emergency approval from the U.S. and other worldwide regulatory agencies are expected to pose little risk to the rare disease community, including to patients with compromised immune systems or those participating in gene therapy studies. That was the message of a recent…