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Duchenne regulatory science consortium meeting on disease progression modeling for duchenne muscular dystrophy

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.

Publication metadata

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


DOI: 10.1371/


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This meeting was funded by the Duchenne Regulatory Science Consortium, which is funded by a grant from Parent Project Muscular Dystrophy