Interested in Registries for Rare Neurological Diseases? Then join our Scientific Symposium on October 10 – online and free of charge!
PROGRAMME
Session 1: Framework for RND Registries
| TIME (CET) | TOPIC | SPEAKER |
| 08:30 | Welcome and overview of ERN-RND activities | Holm Graessner / Ludger Schöls / symposium chairs |
| 08:40 | —– KEYNOTE —- Why Registries Matter for Rare Neurological Diseases: from Natural History to Trial Readiness | Thomas Klockgether, German Centre for Neurodegenerative Diseases (DZNE), University Bonn, Germany |
| 09:00 | Framework for Multistakeholder Patient Registries in the Field of Rare Neurological Diseases | Nicole Wolf, Amsterdam UMC – Amsterdam University Medical Center, Netherlands |
| 09:20 | Patient Perspective: What should registries deliver for patients and families? | tbc |
| 09:30 | Flash talks (5 min max): ERN-RND local registry initiatives | |
| 09:50 | Coffee Break |
Session 2: Registry Examples in ERN-RND
| TIME (CET) | TOPIC | SPEAKER |
| 10:15 | TreatHSP Natural History and Outcome Validation Platform | Rebecca Schüle, University Hospital Heidelberg, Germany |
| 10:30 | The French MSA registry | David Bendetowicz, University Hospital Bordeaux, France |
| 10:45 | NKX2-1 registry: Building an International Disease Registry in a Very Rare Disorder | Dario Ortigoza, Sant Joan de Déu Hospital, Barcelona, Spain |
| 11:00 | Panel Discussion | |
| 11:30 | Coffee Break |
Session 3: Regulatory and Methodological Perspectives
| TIME (CET) | TOPIC | SPEAKER |
| 12:00 | Registry Qualification and Regulatory Expectations for Rare Disease Evidence Generation | Kelly Plueschke, European Medicines Agency |
| 12:30 | Natural History Studies as Historical Control Data in Trials | tbc |
| 12:45 | Using Registries in Phase 4 / Post-Authorisation Studies | tbc |
| 13:00 | Panel Discussion | |
| 13:30 | Closing Remarks and Next Steps |
For further information please contact Sophie Ripp ()
Download programme here.