First European trial site opens at Great Ormond Street Hospital to study prevention of coronary artery aneurysms in Kawasaki Disease.
Kawasaki Disease Coronary Artery Aneurysm Prevention Trial Read More »
MPS Expert Webinar | GENE THERAPY CTs FOR MPS TYPE III July 21th 2021.
MPS Expert Webinar | GENE THERAPY CTs FOR MPS TYPE III Read More »
The commentary ‘Boosting delivery of rare disease therapies: the IRDiRC Orphan Drug Development Guidebook’ has been published in Nature Reviews
IRDIRC Orphan Drug Development Guidebook Read More »
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.
New Free-to-View Live Webinars | May – June 2020 Read More »
Enthusiastic and ambitious Assistant Professor need at Radboud UMC to perform preclinical therapeutic studies for inherited neurometabolic diseases.
Assistant Professor ‘Preclinical therapies for inherited neurometabolic diseases’ Read More »
Three-day preceptorship programme offers specialized training in lysosomal storage diseases management for international physicians.
European Joint Programme on Rare Diseases announces funding opportunities for research mobility and specialized training workshops.
Research Mobility Fellowships and Research Training Workshops Read More »
Annual international campaign raises awareness about rare diseases and their impact on patients’ lives on February 29, 2020.
Rare Disease Day 2020: Get involved Read More »
New research examines availability and accessibility of EMA-approved orphan medicines for hereditary metabolic diseases across Europe.
MetabERN Publication in Orphanet Journal of Rare Diseases Read More »
New initiative explores patient experiences in rare disease care to improve healthcare services and clinical guidelines development.
“Patient Journeys”: improving care by patient involvement Read More »
International congress in Berlin unites scientists, experts and patients to discuss breakthrough research and policies in rare diseases.
The Joint IRDiRC-RE(ACT) Congress | March 11-14, 2020 Read More »
New funding scheme opening in 2020 supports workshops and conferences to strengthen rare disease research networks across Europe.
EJP RD | NETWORKING SUPPORT SCHEME (NSS) Read More »
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