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  1. Article

    Open Access

    Capturing Data in Rare Disease Registries to Support Regulatory Decision Making: A Survey Study Among Industry and Other Stakeholders

    In rare diseases, registry-based studies can be used to provide natural history data pre-approval and complement drug efficacy and/or safety knowledge post-approval.

    Carla J. Jonker, Sieta T. de Vries, H. Marijke van den Berg… in Drug Safety (2021)

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  2. Article

    Open Access

    Different impact of factor VIII products on inhibitor development?

    Inhibitors are the most serious side effect of haemophilia treatment; they occur in 25–30 % of all patients with severe haemophilia A. Over the last 2 decades, conflicting data on the impact of clotting produc...

    H. Marijke van den Berg in Thrombosis Journal (2016)

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