Emergency situations are a major burden for many IEM-patients and their families. Having an emergency letter and knowing how to use it can prevent life-threatening situations and will optimize communications between parents and doctors.
The IMI conect4children (c4c) consortium is pleased to announce that the first site is now open for the ‘Kawasaki Disease Coronary Artery Aneurysm Prevention trial (KD-CAAP)’ study as of 28 September 2020 at Great Ormond Street Hospital NHS Foundation Trust.
The Clinical Patient Management System (CPMS) was launched by the European Commission (Directorate-General for Health and Food Safety – DG SANTE) on November 20th 2017, as a web-based application aimed at supporting the European Reference Networks (ERNs) in improving the diagnosis and treatment of rare or low prevalence complex diseases across national borders of EU Member States.
The COVID-19 pandemic is testing the resilience of robust health systems around the world. This may be impacting you in many different ways, such as creating additional anxiety or exacerbating other medical or therapy issues related to your Inherited Metabolic Diseases (IMD).
Are you interested in calls and training courses of the European Joint Programme on Rare Diseases? Click here to discover what the EJPRD are planning.
The commentary ‘Boosting delivery of rare disease therapies: the IRDiRC Orphan Drug Development Guidebook’ has been published in Nature Reviews
Enhancing the Quality of Life of Persons Affected by Mucopolysaccharide Across Europe. By taking a multidisciplinary healthcare team approach we can better understand how to work with colleagues to support patients with MPS with their everyday healthcare needs, as well as by having a better understanding of the latest treatment options.
May 16th is the World Congenital Disorders of Glycosylation (CDG) Day. The worldwide CDG community joins efforts around this day to put together global awareness and education.
Enthusiastic and ambitious Assistant Professor need at Radboud UMC to perform preclinical therapeutic studies for inherited neurometabolic diseases.
Due to the evolving public health risk associated with the worldwide outbreak of the Coronavirus, we regret to inform you that we have decided to postpone to a new date the Annual MetabERN Board meeting planned for April 2nd-4th, 2020 at the Mercure Hotel Frankfurt Airport.