Therapy Development for Rare Diseases

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.​

• Background

  Rare diseases are largely ignored by pharmaceutical companies with respect to therapy development 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:

  1. Excellent preclinical research on genetics and (cancer) immunology of rare diseases, generating promising leads for therapeutic intervention.
  2. Its organization of care for rare disease patients and connections to care networks, ensuring access to patient groups.
  3. Expertise on innovative clinical trial design for rare diseases
  4. 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.