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

• Main Authors: Karthik Lingineni (Author), Varun Aggarwal (Author), Juan Francisco Morales (Author), Daniela J. Conrado (Author), Diane Corey (Author), Camille Vong (Author), Jackson Burton (Author), Jane Larkindale (Author), Klaus Romero (Author), Stephan Schmidt (Author), Sarah Kim (Author), the Cooperative International Neuromuscular Research Group investigators and Duchenne Regulatory Science Consortium members (Author)
• Format: Book
• Published: Wiley, 2022-03-01T00:00:00Z.
• Subjects: article
• Online Access: Connect to this object online.

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...