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. 2017 Jan 12:9:ecurrents.md.83071bbd728982f2f1073f4950e03586.
doi: 10.1371/currents.md.83071bbd728982f2f1073f4950e03586.

Duchenne Regulatory Science Consortium Meeting on Disease Progression Modeling for Duchenne Muscular Dystrophy

Jane Larkindale, Richard Abresch  1 , Enrique Aviles  2 , Abby Bronson  3 , Janice Chin  4 , Pat Furlong  3 , Heather Gordish-Dressman  5 , Elizabeth Habeeb-Louks  3 , Erik Henricson  6 , Hans Kroger  7 , Charles Lynn  2 , Stephen Lynn  8 , Dana Martin  9 , Glen Nuckolls  10 , William Rooney  11 , Klaus Romero  2 , Lee Sweeney  12 , Krista Vandenborne  13 , Glenn Walter  14 , Jodi Wolff  15 , Brenda Wong  16 , Craig M McDonald  1 , Members Of The Duchenne Regulatory Science Consortium Imaging-Dmd Consortium And The Cinrg Investigators
Affiliations

Duchenne Regulatory Science Consortium Meeting on Disease Progression Modeling for Duchenne Muscular Dystrophy

Jane Larkindale et al. PLoS Curr. .

Abstract

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.

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References

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