European Medicines Agency [EMA]

An example of public-private partnership (DevelopAKUre) from diverse international groups in industry (Sobi), patient organisations (AKU Society and ALCAP), hospitals (Royal Liverpool, Hospital Necker, and National Institute of Rheumatic Diseases), Academia (Universities of Liverpool and Siena, Institute of Molecular Physiology and Genetics) and Small and Medium Enterprises –SME– (Nordic Bioscience, PSR and Cudos) to cure alkaptonuria (AKU).

A collaboration of Genzyme, International Pompe Association (IPA); UK, Dutch, and US Pompe patient organisations, Erasmus Medical Center and Duke University to establish appropriate measures to ensure the data in clinical trials is robust and will satisfy regulatory and payer requirements.