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AFM Telethon

Sponsors » Biomarin

Satellite symposium sponsored by BioMarin

Lessons learned from DMD natural history for drisapersen clinical trials

Tuesday, 15 March 2016
18/00-18:30 Welcome cocktail
18:30-20:00 hrs
Room Pasteur


Faculty:
Chairman: Dr. Laurent Servais, Paris, France
Dr. Giles Campion, London, UK
Dr. Pierre Carlier, Paris, France
Prof. Nathalie M. Goemans, Leuven, Belgium


Programme:

18:30-18:40 Introduction
Giles Campion, BioMarin London, UK

18:40-18:50 What did we learn from natural history trials in DMD?
Nathalie M. Goemans, Leuven, Belgium

18:50-19:05 Overview of (long-term) efficacy and safety of drisapersen in DMD clinical trials
Laurent Servais, Paris, France

19:05-19:20 What lessons learned from natural history trials in DMD can be used to interpret drisapersen clinical trial outcomes?
Laurent Servais, Paris, France

19:20-19:35 Lessons learned from clinical trials in DMD: MRI as an outcome measure
Pierre Carlier, Paris, France

19:35:19:45 How can future drisapersen clinical trials broaden its application in DMD?
Giles Campion, BioMarin London, UK

19:45-20:00 Panel discussion based on questions from the audience
All; moderated by Laurent Servais, Paris, France

 

During this BioMarin-sponsored satellite symposium, the following topics will be addressed and discussed:
• What did we learn from DMD natural history trials with regard to the predictors of clinical progression?
• What were the (long-term) efficacy and safety results of the existing drisapersen clinical trials?
• How do the lessons learned from the natural history trials impact the interpretation of the existing drisapersen clinical trial results?
• How may the lessons learned from the natural history trials and drisapersen clinical trials impact the interpretation of other trials in DMD?
• What did we learn from DMD clinical trials with regard to magnetic resonance imaging (MRI) as a new outcome measure and how should we most optimally apply MRI as an outcome measure in DMD?
• How should we design future drisapersen clinical trials in the DMD population?