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.
The NCL Foundation will evaluate proposals for its NCL Research Award. The goal of this award is to support an innovative pilot project at the Postdoctoral fellowship level. The NCL Foundation encourages junior scientists, clinical researchers and medical fellows worldwide to submit project proposals that hold promise to help find or to facilitate the moving forward of therapies for CLN3 disease.
Three key documents are now available for candidates to prepare their applications for the Call for membership to existing ERNs. The first call for new members to join existing 24 ERNs is going to be launched on 30 of September 2019 until 30 of November 2019.
Malta has designated the Mater Dei Hospital (MDH) as a National Coordination Hub for ERNs and MetabERN has been nominated by six associated national centres.
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