Symposium - Clinical trials: The bridge to an optimal registration

Tuesday, October 29, 2019
Leiden University Medical Center
Room 2 - Building 1, Leiden

Find the gaps together with us at the ProPharma Group Centre of Excellence; a life cycle approach


Will your clinical development in the future deliver new treatment options? Solid science will always be a foundation, but a successful development process stretches beyond scientific opportunities. Find the gaps and apply a life cycle approach for product development.

Professor Doctor Annemieke Aartsma-Rus will inform you on the lessons learned from a failure of a registration trial on a Duchenne muscular dystrophy treatment.

Step into the world of Phase II studies based on a multidisciplinary approach and strategy building. Discuss topics as dose finding, comparator picking, epidemiology, and dosing formulations. Focus on small (Phase II/III) studies on rare diseases.


16.30              Doors open
17.00              Welcome
17.15 – 18.15 Annemieke Aartsma-Rus (Professor of translational genetics, LUMC),
                       Therapy development for rare diseases takes more than a therapeutic
18.15 – 19.00 Thomas de Vlaam (Founder and CEO, Amylon Therapeutics): Industry
19.00 – 19.30 ProPharma Group: Regulatory affairs
19.30              Drinks & Networking
20.30              Close

Register for free at

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