PCR29 Progressive Disease Milestones and Survival in Duchenne Muscular Dystrophy (DMD): A Model-Based Synthesis for Extrapolating Lifetime Treatment Effects From Clinical Trial Results
Abstract
Authors
E. Smith N. Posner J. Signorovitch M. Johnson V. Merla A. Gomez-Lievano A. Zhang A. Sharma M. Inês J. Cappelleri P. Cislo