Haute Autorité de Santé« Back to Glossary Index
« Back to Glossary Index
- Glossary: Patient-reported outcome
- Patients Involved - DevelopAKUre for AKUAn 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).
- Patients Involved - Duchenne Parent ProjectA patients involved case report on the Duchenne Parent Project collaborating with academia and industry to find a cure/treatment for Duchenne Muscular Dystrophy.
- Patients Involved - Phase III Hepatitis CA collaboration between Bristol-Myers Squibb (BMS) with FUNDHEPA (patient organisation) to give more patients the opportunity to receive an innovative treatment for Hepatitis C through the participation in a clinical trial.
- Patients Involved - An ultra orphan diseaseA 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.