A team of MetabERN members has written a publication in the Orphanet Journal of Rare Diseases (OJRD) titled “Availability, accessibility and delivery to patients of the 28 orphan medicines approved by the European Medicines Agency (EMA) for hereditary metabolic diseases in the MetabERN network”. The European Medicine Agency granted marketing approval to 164 orphan medicinal […]
Since the end of 2019, MetabERN has 9 new Healthcare Providers from 5 different countries that are Affiliated Partners (APs) to our network. They are bringing the total number of HCPs that are involved in MetabERN to 78 from 23 EU members states. The names and contact details of these HCPs can be found on this page of this website
Patient Journeys “I will not be ashamed to say ‘I don’t know’, nor will I fail to call in my colleagues…” But what if there are no other healthcare...
On-time diagnosis is crucial for access to treatment and clinical trials, disease management, and to access the proper care services at the earliest of the disease possible.
This joint event will continue the RE(ACT) Congress series (6th edition) and IRDiRC Conference series (4th edition). It aims to bring together scientific leaders and experts and young scientists from a variety of breakthrough scientific fields to present cutting-edge research, exchange ideas, and discuss policies related to rare diseases research. Patients and patient organizations, who are committed to research, will also be in attendance to share their experiences and perspectives.
16th International SPDM Symposium | 19-20 March 2020
European Working Group on Gaucher Disease Webinar | Laboratory Diagnostics of Gaucher Disease - 11 Dec 2019 from 17:00-18:00 CET
Patients and specialists of Anderson-Fabry disease will meet in Florence on March 28th-29th, for the Annual Italian Meeting organized by AIAF Onlus.
A Networking Support Scheme (NSS) is implemented to encourage the sharing of knowledge on rare diseases and/or rare cancers of healthcare professionals, researchers, and patient advocacy organizations.
The ERA-Net E-Rare has successfully implemented ten Joint Transnational Calls for rare disease research projects since 2006.