Use of funded multicenter prospective longitudinal databases to inform clinical trials in rare diseases—Examination of cholestatic liver disease in Alagille syndrome

The conduct of long‐term conventional randomized clinical trials in rare diseases is very difficult, making evidenced‐based drug development problematic. As a result, real‐world data/evidence are being used more frequently to assess new therapeutic approaches in orphan diseases. In this investigatio...

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Detalles Bibliográficos
Autores principales: Shneider, Benjamin L., Kamath, Binita M., Magee, John C., Goodrich, Nathan P., Loomes, Kathleen M., Ye, Wen, Spino, Cathie, Alonso, Estella M., Molleston, Jean P., Bezerra, Jorge A., Wang, Kasper S., Karpen, Saul J., Horslen, Simon P., Guthery, Stephen L., Rosenthal, Philip, Squires, Robert H., Sokol, Ronald J.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: John Wiley and Sons Inc. 2022
Materias:
Original Articles
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9315119/
https://www.ncbi.nlm.nih.gov/pubmed/35506349
http://dx.doi.org/10.1002/hep4.1970

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