Wednesday, June 5, 2019​

16:00-18:00 Top Challenges Facing the Pharmaceutical Industry in Down syndrome Drug Development

The Market: Is it large enough to justify drug development? (Pier-Vincenzo Piazza, AELIS Pharma)

Translationality: Are we using the most adequate tools in the evaluation of drug effects in the transfer from preclinical studies to clinical trials

Mouse-based genetic modeling of Down syndrome. (William C Mobley, USC San Diego)

Bordeaux Maze Test  (Rafael de la Torre, IMIM)

Clinical Trials Outcomes: Difficulties in finding primary outcomes acceptable for regulatory authorities (FDA, EMA)

The NIH Toolbox Cognitive Battery for intellectual disabilities (Len Abbeduto, MIND Institute)

Regulatory Aspects: The EMA perspective in drug development of orphan drugs targeting neurodevelopmental disorders (Fernando Mendez, EMA Committee for Orphan Medicinal Products)


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Registration cost: 25 €