How can we effectively translate promising therapies from concept to patient care? On September 24 we will host a symposium in which we will explore the latest developments in therapy development for rare diseases — from innovative cell and gene therapies to drug repurposing and personalized (n=1) treatments.
Drawing on Radboudumc projects, we will highlight key opportunities, challenges, and the essential steps needed to translate (pre)clinical research into sustainable and accessible treatment options.
The symposium will also offer plenty of opportunities to connect with colleagues, exchange knowledge and experiences, and explore which next steps may be most relevant for your own research or projects.
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13:00 – 13:30 | Therapy development for rare diseases: from scientific discovery to patient impact Dirk Lefeber and Saskia de Wildt
13:30 – 14:00 | Creating new possibilities: the cell & gene therapy journey Renée Buurman-Raaijmakers and Gerty Schreibelt
14:00 – 14:30 | Tailor-made antisense therapies Rob Collin and Karin Ruijtenbeek
14:30 – 15:00 | Networking Break
15:00 – 15:30 | The power of drug repurposing Carine van der Vleuten, Edith Klappe and Rob ter Heine
15:30 – 16:00 | Facilitating Sustainable Access for Patients Sibren van den Berg (RARE-NL) and Maaike Oosterveer
16:00 – 16:45 | Moving forward: practical steps to accelerate your therapy development project Interactive session with all participants
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24 September 2026, 13:00 - 17:00h
Location: Lammerszaal, route 86
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You can register via this form.
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For questions about the event, please contact: marga.bouma@radboudumc.nl