Innovative therapies, such as mRNA, gene therapy and CAR-T cell therapy, have enormous potential for treating children with rare diseases. The development, early-stage clinical trials, regulation and accessibility of these therapies require collaboration and new approaches. During this symposium, we will highlight developments inside and outside academic hospitals and aim to discuss the challenges and opportunities with all experts involved.
This symposium is in Dutch. The programme will follow soon.
We have made the presentations available of the symposium of 25 January 2025.
Download the presentations (in Dutch)