News

Organizations Rally to Help Ukrainian Rare Disease Patients

A Russian military plane crash near Tetiana Zamorska’s home in Kyiv, Ukraine, was a sign that it was time for her and her family to leave. The treacherous, 34-hour pilgrimage that ultimately brought the group of eight by car to temporary accommodations in neighboring Poland last month was physically and emotionally difficult,…

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…

Edsivo Named Breakthrough Therapy by FDA for COL3A1-positive vEDS

Edsivo (celiprolol) has been granted a breakthrough therapy designation for COL3A1-positive vascular Ehlers-Danlos syndrome (vEDS) by the U.S. Food and Drug Administration (FDA). The designation is meant to expedite the development and review of medicines that are intended to treat a serious condition, and for which early clinical…

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…

Lower Quality of Life Found in Young Women With Hypermobility

Orthostatic intolerance — a condition characterized by symptoms like dizziness, elevated heart rate, or blurred vision upon moving to an upright position — was common, and had a significant impact, among young women and adolescent girls with hypermobile Ehlers-Danlos syndrome (hEDS) and generalized hypermobility spectrum disorder (G-HSD), according…