50 Million euro investment for LUMC spin-off RougeTx advances therapies for inherited bleeding disorders.
On Saturday, October 18, Leiden University Medical Center (LUMC) hosted a national day dedicated to patients affected by the rare genetic vascular disorder ROW/HHT. This event brought together patients, families, clinicians, and researchers around a shared goal: to exchange on scientific advances and strengthen the link between science and patients. The day was organized by the team of reNEW AI Franck Lebrin in collaboration with the ROW/HHT Nederland Foundation, with contributions from many other reNEW researchers.
The day centered around the rare condition Rendu Osler Weber (ROW), also known as Hereditary Hemorrhagic Telangiectasia (HHT). Patients that suffer from this genetic vascular disorder have a defect in their vessel walls which leads to abnormal connections between arteries and veins – arteriovenous malformations. Patients suffer from frequent bleeding, primarily nosebleeds but potentially also in internal organs.
reNEW PI Christine Mummery opened the official program by welcoming the attendees. She then provided an overview of the major achievements of ROW/HHT research at LUMC and emphasized the importance of active patient participation in the process of medical innovation.
The program then introduced a new format of interactive small-group workshops, focused on key topics such as developing drugs for rare diseases, new therapeutic approaches including gene therapy, and innovations in ultrasound imaging from the International JOINTLAB INSERM between LUMC and the Institute of Physics for Medicine Paris, dedicated to diagnosis and treatment monitoring. A dedicated workshop explored how patients contribute to setting scientific priorities, designing clinical studies, and sharing results.
These sessions, led by young researchers, PhD students, and postdocs, encouraged lively exchanges between science and patient experience. The 5-minute presentation / 20-minute discussion format fostered open and constructive dialogue, truly placing the patient at the center of the conversation.
A highlight of the day was the “Life Stories” session, moderated by Dr. Hans-Jurgen Mager (St. Antonius Hospital, Utrecht), featuring patients Amy Herl and Liemarvin Bonevacia, a Dutch Olympic athlete living with ROW/HHT. They shared their personal journeys and daily challenges, offering moving and hopeful testimonies that reminded everyone of the deeply human side of research. We warmly thank all participants for their engagement. This day marks the beginning of a new dynamic between science and patient experience, dedicated to advancing understanding and treatment of ROW/HHT.
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This event and the involved researchers were supported by amongst others the Novo Nordisk Center for Stem Cell Medicine, reNEW and the European program EIC Pathfinder Microvasc. The event reflects commitment of reNEW and Europe to strengthening collaborative research and patient involvement.
