News

An insightful analysis of five common myths about orphan drugs and their real impact on healthcare systems and rare disease treatment.

The members of the “conect4children” (c4c) initiative today announced the start of a large collaborative paediatric network that will facilitate the development of new drugs and other therapies for the entire paediatric population in Europe.

Chafea organised a kick-off meeting for five new rare diseases’ registries in Luxembourg on the 17th of April 2018, which involved 40 participants.

Health Commissioner Andriukaitis evaluates the first year of ERN operations and outlines key challenges for rare disease networks.

A critical request for blood and urine samples from metabolic disorder patients to maintain high quality diagnostic laboratory standards.

In February 2018, a European Parliamentary Meeting gathered healthcare leaders to improve diagnostic timelines for rare diseases.

Historical position statement from 24 ERN coordinators emphasizing the crucial need for European collaboration in rare disease treatment.

French-Canadian research team discovers “epi-cblC,” changing our understanding of how rare metabolic diseases can be inherited.

MetabERN achieved its first virtual patient consultation via the Clinical Patient Management System, marking a new era in rare disease care.

MetabERN’s first meeting with Patient and Family Associations established a framework for collaborative rare disease care development.

The European Commission’s launch of the Clinical Patient Management System created a new framework for cross-border rare disease care.