In the framework of the EJP-RD project (European Joint Programme on Rare Diseases), there are two calls for funding opened on 3 February: 1. Research Mobility Fellowships and 2. Research Training Workshops Target group (persons affiliated to ERN Full Members or Affiliated Partners) : 1. PhD students and young Medical Doctors (having finished their first […]
Thus far 11 members of MetabERN and one HCP which is not part of MetabERN have joined the project and more than 500 patient records have been added to the registry (see directly on the homepage of the registry: https://www.u-imd-registry.org/). While this is a great success, U-IMD is still in need of wider participation of the ERN to reach all of its aims.
Rare Disease Day 2020 takes place on the last day of February. This year is the 13th edition of the international day of rare disease awareness, and highlights that 'Rare is many, rare is strong and rare is proud'. The main objective of Rare Disease Day is to raise awareness amongst the general public and decision-makers about rare diseases and their impact on patients' lives.
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...
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.
The goal of this meeting was to encourage adult MPS patients to be independent – Stand on their Own.
Sant Joan de Deu Hospital (Barcelona, Spain), one of the 69 MetabERN healthcare providers, has opened a clinical trial that recruits patients for an open-label study in male pediatric patients with cerebral X-LINKED ADRENOLEUKODYSTROPHY (cALD) to assess the effect of a new experimental treatment on the progression of cerebral lesions.