Development of a model‐based clinical trial simulation platform to optimize the design of clinical trials for Duchenne muscular dystrophy

Development of a model‐based clinical trial simulation platform to optimize the design of clinical trials for Duchenne muscular dystrophy

Abstract Early clinical trials of therapies to treat Duchenne muscular dystrophy (DMD), a fatal genetic X‐linked pediatric disease, have been designed based on the limited understanding of natural disease progression and variability in clinical measures over different stages of the continuum of the...

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Bibliographic Details
Main Authors: Karthik Lingineni, Varun Aggarwal, Juan Francisco Morales, Daniela J. Conrado, Diane Corey, Camille Vong, Jackson Burton, Jane Larkindale, Klaus Romero, Stephan Schmidt, Sarah Kim, the Cooperative International Neuromuscular Research Group investigators and Duchenne Regulatory Science Consortium members
Format: Article
Language:English
Published: Wiley 2022-03-01
Series:CPT: Pharmacometrics & Systems Pharmacology
Online Access:https://doi.org/10.1002/psp4.12753

Internet

https://doi.org/10.1002/psp4.12753

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