Access to medicines for rare diseases: A European regulatory roadmap for academia

• Main Authors: Noa Rosenberg (Author), Sibren van den Berg (Author), Nina N. Stolwijk (Author), Bart A. W. Jacobs (Author), Hendrika C. Post (Author), Anna M. G. Pasmooij (Author), Saco J. de Visser (Author), Carla E. M. Hollak (Author)
• Format: Book
• Published: Frontiers Media S.A., 2023-02-01T00:00:00Z.
• Subjects: article
• Online Access: Connect to this object online.

Background: Novel or repurposed medicines for rare diseases often emerge from fundamental research or empirical findings in academia. However, researchers may be insufficiently aware of the possibilities and requirements to bring novel medicinal treatment options to the patient. This paper aims to p...