Dr. Marc Engelen

Host: prof. dr. Marjo van der Knaap Departement of Child Neurology, Amsterdam Leukodystrophy Center, Emma Children’s Hospital
Amsterdam University Medical Centers.

Guest speaker: Marc Engelen
(Pediatric) Neurologist/Principal Investigator Amsterdam Leukodystrophy Center Department of (Pediatric) Neurology.

Title: Clinical trials in rare diseases.

Abstract:

Clinical trials are challenging, especially in rare diseases. Knowledge gaps in natural history make it difficult to select appropriate endpoints for clinical trials. Furthermore, clinimetric tools to quantify these endpoints are often not accurate and sensitive. This means long trials with many patients, often not feasible. Since therapeutic options are emerging, it is highly important to improve our ability test efficacy of potential new treatments effectively. The presentation will illustrate these concepts and solutions using X-linked adrenoleukodystrophy (ALD) as a model.

Short Bio:

Marc Engelen obtained an MD (2002) and PhD (2012) from the University of Amsterdam. He is currently a (pediatric) neurologist at Amsterdam UMC. As part of the Amsterdam Leukodystrophy Center he is involved in leukodystrophy research. One of his interests is improving clinical trials for rare diseases by defining better (surrogate) outcome measures, and that will be the subject of the presentation today.