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

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...

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Detalles Bibliográficos
Autores principales: Rosenberg, Noa, van den Berg, Sibren, Stolwijk, Nina N., Jacobs, Bart A. W., Post, Hendrika C., Pasmooij, Anna M. G., de Visser, Saco J., Hollak, Carla E. M.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Frontiers Media S.A. 2023
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10012277/
https://www.ncbi.nlm.nih.gov/pubmed/36925633
http://dx.doi.org/10.3389/fphar.2023.1142351