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

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

Abstract 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 inv...

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Main Authors: Benjamin L. Shneider, Binita M. Kamath, John C. Magee, Nathan P. Goodrich, Kathleen M. Loomes, Wen Ye, Cathie Spino, Estella M. Alonso, Jean P. Molleston, Jorge A. Bezerra, Kasper S. Wang, Saul J. Karpen, Simon P. Horslen, Stephen L. Guthery, Philip Rosenthal, Robert H. Squires, Ronald J. Sokol, for the Childhood Liver Disease Research Network (ChiLDReN)
Format: Article
Language:English
Published: Wolters Kluwer Health/LWW 2022-08-01
Series:Hepatology Communications
Online Access:https://doi.org/10.1002/hep4.1970

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https://doi.org/10.1002/hep4.1970

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