Rare Diseases

World’s first gene therapy for p47 Chronic Granulomatous Disease developed by UCL and GOSH

UCL and Great Ormond Street Hospital have developed the world's first gene therapy for p47 Chronic Granulomatous Disease.

The provision of effective cross-border healthcare

Professor Ruth Ladenstein from SIOPE (the European Society for Paediatric Oncology) discusses how ERN PaedCan facilitates effective cross-border healthcare The European Commission launched 24 different...

Modern research into rare diseases

Research infrastructure project manager at EURORDIS, Virginie Bros-Facer highlights the extent of rare diseases in Europe in this insightful article

Health officials pledge to reduce cholera deaths by 90%

Health Officials from across the globe are set to meet in France and pledge to dramatically reduce cholera deaths by 2030

The impact of clinical research in rare diseases

Kit C.B. Roes from the University Medical Centre, Utrecht outlines why clinical research in rare diseases requires swift implementation of new methodology

The European Confederation of Pharmaceutical Entrepreneurs AISBL

The European Confederation of Pharmaceutical Entrepreneurs (EUCOPE) is Europe’s principal trade body for small to medium-sized innovative companies working in the field of pharmaceuticals....

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Latest Academic Articles

The latest academic articles from key research stakeholders