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

members of the Duchenne Regulatory Science Consortium, Imaging-DMD Consortium and the CINRG Investigators

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Abstract

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.

Original languageEnglish (US)
JournalPLoS Currents
Volume8
Issue numberMuscularDystrophy
Publication statusPublished - 2016

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ASJC Scopus subject areas

  • Medicine (miscellaneous)

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members of the Duchenne Regulatory Science Consortium, Imaging-DMD Consortium and the CINRG Investigators (2016). Duchenne regulatory science consortium meeting on disease progression modeling for duchenne muscular dystrophy. PLoS Currents, 8(MuscularDystrophy).