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Lookup NU author(s): Dr Stephen Lynn
This work is licensed under a Creative Commons Attribution 4.0 International License (CC BY 4.0).
© 2016, Public Library of Science. All rights reserved. Introduction: The Duchenne Regulatory Science Consortium (D-RSC) was established to develop tools to accelerate drug development for DMD. The resulting tools are anticipated to meet validity requirements outlined by qualification/endorsement pathways at both the U.S. Food and Drug Administration (FDA) and European Medicines Administration (EMA), and will be made available to the drug development community. The initial goals of the consortium include the development of a disease progression model, with the goal of creating a model that would be used to forecast changes in clinically meaningful endpoints, which would inform clinical trial protocol development and data analysis. Methods: In April of 2016 the consortium and other experts met to formulate plans for the development of the model. Conclusions: Here we report the results of the meeting, and discussion as to the form of the model that we plan to move forward to develop, after input from the regulatory authorities.
Author(s): Larkindale J, Abresch R, Aviles E, Bronson A, Chin J, Furlong P, Gordish-Dressman H, Habeeb-Louks E, Henricson E, Kroger H, Lynn C, Lynn S, Martin D, Nuckolls G, Rooney W, Romero K, Sweeney L, Vandenborne K, Walter G, Wolff J, Wong B, McDonald CM
Publication type: Article
Publication status: Published
Journal: PLOS Currents: Muscular Dystrophy
Year: 2017
Volume: 8
Online publication date: 12/01/2017
Acceptance date: 02/04/2016
Date deposited: 21/04/2017
ISSN (electronic): 2157-3999
Publisher: Public Library of Science
URL: http://currents.plos.org/md/article/duchenne-regulatory-science-consortium-meeting-on-disease-progression-modeling-for-duchenne-muscular-dystrophy/
DOI: 10.1371/currents.md.83071bbd728982f2f1073f4950e03586
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