Exciting news for researchers, clinicians, and healthcare professionals! The eagerly anticipated MOOC, « From Lab to Clinic: Translational Research for Rare Diseases » is launching on April 17th. This 5-week course offers a unique opportunity to gain insight into the issues, challenges, and opportunities in translating research into treatments for rare disease patients. With just 4 hours…
IRDiRC’s Therapies Scientific Committee (TSC) would like to broaden the committee membership, to represent the worldwide rare diseases therapies community in its full width, from early innovation towards regulatory approval and therapeutic support of patients. As such, the TSC has three openings for new members, specifically encouraging participants from the following regions: Africa, Asia, Australasia or South America.…
In the months of May and June 2022, the European Medicines Agency (EMA) recommended granting various forms of marketing authorisation in the European Union (EU) for the following rare disease-targeted therapies: First therapy to treat two types of Niemann-Pick disease, a rare genetic metabolic disorder: Xenpozyme (olipudase alfa), a therapy for the treatment of non-central…
The ProDGNE consortium, funded as part of the EJP RD’s Joint Transnational Call 2020 « Pre-Clinical Research to Develop Effective Therapies for Rare Diseases », announces its ProDGNE Kick-Off Webinar. The webinar will take place on June 03, 2021 from 18.00 – 19.00 CET. The webinar will present ProDGNE, a 3-year transnational pre-clinical research project that brings together patients and researchers from…