PhD candidate Sophie Hordijk has been selected as one of the five recipients of the Professor Heimburger Award 2025, presented by global biotechnology company CSL Behring. This award, which includes a €25,000 grant, celebrates outstanding clinical and preclinical research by early-career scientists working to improve care for patients with bleeding disorders.
A major clinical challenge in von Willebrand disease is gastrointestinal (GI) bleeding, often caused by angiodysplasia, a severe vascular manifestation of the disease. Despite its impact, the molecular mechanisms behind angiodysplasia remain poorly understood. The proposal builds on work recently published in Blood, in which MADD, a guanine nucleotide exchange factor, was identified as a key regulator of Weibel-Palade body exocytosis. Patients with mutations in MADD show severely reduced Von Willebrand factor levels. Proteomic analysis further revealed downregulation of Neural Proliferation, Differentiation, and Control 1 (NPDC1). The function of this protein in endothelial cells remains uncharacterized, though it has been implicated in other systems in cell cycle regulation, differentiation, and retinoid signaling, which are pathways linked to angiogenesis.
Sophie will therefore explore the role of NPDC1 in VWF secretion and vascular development, aiming to generate new insights to support the development of therapeutics for both rare and common vascular diseases.
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