News

Europe Awaits Proposed New Framework for Sharing Health Data

The European Commission is expected to propose a new governing framework for health data next month, called the European Health Data Space (EHDS), with the aim of connecting national health systems to facilitate secure and efficient transfer of data across systems in different European nations. The move is expected to…

US Department of Defense Opening FA Research to Grant Awards

The U.S. Department of Defense (DoD) is offering grant awards to researchers working to improve care for Friedreich’s ataxia. The DoD’s Congressionally Directed Medical Research Programs (CDMRP) is a funding organization whose goal is to further the development of new approaches to address specific areas of biomedical research as…

Elamipretide Wins FDA Orphan Drug Status, Phase 2 Trial Starts

Stealth BioTherapeutics’ lead candidate elamipretide has been granted orphan drug status by the U.S. Food and Drug Administration (FDA) for the treatment of Friedreich’s ataxia (FA). An orphan drug designation seeks to encourage therapies for rare diseases, through benefits such as seven years of market exclusivity upon approval…

Increased Education Crucial to Improving Rare Disease Care, Survey Finds

Healthcare providers involved in diagnosing and treating rare diseases believe that increased physician education and collaboration with specialized facilities will have the greatest positive impact on treating these conditions over the next five years, according to results from a 2021 survey. Definitive Healthcare, a healthcare commercial intelligence company, conducted…

Thinning of Eye’s Retina May Be Useful Marker of FA Progression

Reduced thickness of the peripapillary retinal nerve fiber layer (RNFL) in the eye’s retina is associated with worse disease severity in people with Friedreich’s ataxia (FA), an Irish study concluded. Follow-up measurements showed a thinning in this layer was linked to worsening disease severity, suggesting that regular eye assessments may…