About Therapy development for rare diseases
At Radboudumc, we are dedicated to developing innovative therapies for rare diseases. By combining cutting-edge fundamental research with clinical expertise, and working closely with patients and partners, we enable the rapid translation of promising discoveries into effective treatments.
read moreAbout Therapy development for rare diseases
At Radboudumc, we are dedicated to developing innovative therapies for rare diseases. By combining cutting-edge fundamental research with clinical expertise, and working closely with patients and partners, we enable the rapid translation of promising discoveries into effective treatments.
-
Rare diseases are very challenging for classical therapy development in view of the low patient numbers, yet frequently pose a huge burden on the quality of life of patients and their families. The Radboudumc is excellently positioned to take a leading role in the development and application of personalized therapies for rare diseases, because of:
- Excellent preclinical research on genetics and (cancer) immunology of rare diseases, generating promising leads for therapeutic intervention.
- Its organization of care for rare disease patients and connections to care networks, ensuring access to patient groups.
- Expertise on innovative clinical trial design for rare diseases
- Availability of infrastructure (i.e. GMP facility) for and knowledge on development and production of advanced therapy medicinal products (ATMPs), which include cell- and gene therapy products.
Meet our researchers and their research
More information coming soon.
Explore training and education
-
There is a significant need to train (Biomedical Sciences) students in developing drug, cell- and gene-based therapies from a preclinical, clinical, and regulatory perspective. To address this need, a new BMS master specialisation ‘Therapy Development’ will be developed.
About the specialisation
Developing therapies for rare diseases requires a comprehensive understanding of advanced science and technology as well as the complexities that these diseases present. The new specialisation covers all aspects of traditional therapy development, with an accent on tailoring this for rare diseases. With this specialisation, we aim to create a clear path for students interested in therapy development. This will additionally help in strengthening the capacity within Radboudumc to train professionals who can connect research, drug development and valorisation to accelerate the advancement of drug, cell- and gene-based therapies. More information on Development of Cell, Drug and Gene Therapies | Radboud University.
BMS Master’s programme
Do you want to know more about the Biomedical Sciences Master’s programme? Check this webpage.
-
More information about this specific topic will follow soon.
Access research infrastructure and partnerships
We collaborate national and international with several initiatives and can help you get in contact with these initiatives. More information is coming soon.