Author Archives: Forest Ray PhD

Oxford-Harrington Partnership Targets FA as First Priority

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…

Study Will Test Using Calcitriol to Boost Frataxin

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…

Potential Gene Therapy for Heart Disease in FA Gets FDA Support

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…

FARA to FA Patients: Consider Enrolling in 3 Clinical Trials

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…

Healx Joins With FARA, Ataxia UK for Work on Potential FA Therapies

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 —…

74,000 Sign FARA Petition Asking FDA, Reata to Move Ahead on Omav

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…

FA Research Alliance Petitioning for Urgent Access to Omaveloxolone

With backing from a petition that is now circulating online, the Friedrich’s Ataxia Research Alliance (FARA) will ask Reata Pharmaceuticals and the U.S. Food and Drug Administration to make omaveloxolone (RTA 408) available to people living with Friedrich’s ataxia (FA) — for whom no approved treatments currently exist.

Clinical Studies Continue to Recruit Individuals With FA

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…

Google’s Project Euphonia Asking Patients to Help With Speech Recognition Technology

Team Gleason and CureDuchenne have teamed with Google’s Project Euphonia to make automated speech recognition more accessible to people with neurological disorders, including Friedreich’s ataxia. Although automated speech recognition increasingly powers voice-activated technologies like Google Assistant, it struggles to adapt to changes in speech that accompany certain…

Gene Editing Seen to Restore Frataxin Level in Cells From Patients

The CRISPR-Cas9 gene editing technique safely removed the gene expansion that causes Friedrich’s ataxia (FA), allowing for normal frataxin levels and more functional mitochondria in cells taken from patients, an early study reported. This work, and further experiments in mice, supports the potential of a stem cell approach to treating…