SESSION 1 - EUROPEAN REFERENCE NETWORK REGISTRIES 4 Videos Rare Disease Research Catalyst Data Overview 9:02 European Registries for Rare Endocrine and Bone 15:18 Epidermolysis Bullosa (EB) Registry 11:16 ERN Lung Registries and Academy 9:00 SESSION 2 - CLINICAL TRIALS STAKEHOLDERS' PERSPECTIVES 3 Videos Regulatory 14:50 Public and Patient Involvement (PPI) 15:50 Industry 10:30 SESSION 3 - TRIALS METHODOLOGY AND TRANSLATION 5 Videos Translating Basic Science into Meaningful Therapies 24:23 Platform Trials 15:42 Study Within a Trial (SWAT) 14:23 Clinical Trial Identification Project Overview 6:07 Keynote - Clinical Trial Designs: New Options for the Study of Rare Diseases 1:10:12 CLINICAL TRIAL SPOTLIGHTS 4 Videos Clinical Trial Identification Project Overview 6:07 Autoimmune Pulmonary Alveolar Proteinosis (IMPALA 2) 12:26 Rare Bone Disorders (TOPaZ/ZiPP-LTE) 12:17 Duchenne Muscular Dystrophy, DYNE-DELIVER 17:24
