ECZA-ERN meeting: Advancing therapy development for rare diseases

How can we accelerate the development of therapies for rare diseases? On October 1, the semiannual meeting for Radboudumc’s Centers of Expertise for Rare Diseases (ECZAs) and European Reference Networks (ERNs) will focus on new treatments, clinical trial readiness, and what is needed to bring promising therapies into patient care.

• Program

  • New treatments for rare diseases: recent developments, challenges, and future perspectives, presented by Dirk Lefeber, Chair of the Therapy Accelerator for Rare Diseases.
  • Therapy development within the ECZAs: two practical examples, including repurposing sildenafil for Leigh syndrome and access to sirolimus for vascular anomalies, presented by Mirian Janssen and Carine van der Vleuten.
  • World Café: discussions on developing therapies for patients, preparing ECZAs for future clinical trials, and introducing new medicines at Radboudumc.
  • The meeting concludes with feedback from the discussion tables and key take-home message

  For full program, download flyer (in Dutch)

• When & where

  01 October 2026, 16:00 – 18:00hrs, Oranjezaal, route 607, Radboudumc

• Registration

  No registration required