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

Jane Larkindale; Richard Abresch; Enrique Aviles; Abby Bronson; Janice Chin; Pat Furlong; Heather Gordish-Dressman; Elizabeth Habeeb-Louks; Erik Henricson; Hans Kroger; Charles Lynn; Stephen Lynn; Dana Martin; Glen Nuckolls; William Rooney; Klaus Romero; Lee Sweeney; Krista Vandenborne; Glenn Walter; Jodi Wolff; Brenda Wong; Craig M McDonald; Members Of The Duchenne Regulatory Science Consortium Imaging-Dmd Consortium And The Cinrg Investigators

doi:10.1371/currents.md.83071bbd728982f2f1073f4950e03586

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

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

Authors

Jane Larkindale, Richard Abresch¹, Enrique Aviles², Abby Bronson³, Janice Chin⁴, Pat Furlong³, Heather Gordish-Dressman⁵, Elizabeth Habeeb-Louks³, Erik Henricson⁶, Hans Kroger⁷, Charles Lynn², Stephen Lynn⁸, Dana Martin⁹, Glen Nuckolls¹⁰, William Rooney¹¹, Klaus Romero², Lee Sweeney¹², Krista Vandenborne¹³, Glenn Walter¹⁴, Jodi Wolff¹⁵, Brenda Wong¹⁶, Craig M McDonald¹, Members Of The Duchenne Regulatory Science Consortium Imaging-Dmd Consortium And The Cinrg Investigators

Affiliations

¹ Department of Physical Medicine and Rehabilitation, University of California, Sacramento, California, USA.
² Duchenne Regulatory Science Consortium, Critical Path Institute, Tucson, Arizona, USA.
³ Parent Project Muscular Dystrophy, Hackensack, New Jersey, USA.
⁴ Pfizer, Rare Disease Research Unit, Cambridge, Massachusetts, USA.
⁵ Children's National Medical Center, Research Center for Genetic Medicine, Washington, DC, USA.
⁶ University of California Davis Medical Center, Department of Physical Medicine and Rehabilitation, Sacramento, California, USA.
⁷ PTC Therapeutics, South Plainfield, New Jersey, USA.
⁸ Stephen Lynn, Newcastle University, Newcastle upon Tyne, UK.
⁹ Sarepta Therapeutics, Medical Affairs & Patient Advocacy, Cambridge, Massachusetts, USA.
¹⁰ NINDS, National Institute of Health, Washington DC, USA.
¹¹ Advanced Imaging Research Center, Oregon Health & Science University, Portland, Oregon, USA.
¹² Department of Pharmacology and Therapeutics, University of Florida, Gainesville, Florida, USA.
¹³ Department of Physical Therapy, University of Florida, Gainesville, Florida, USA.
¹⁴ Department of Physiology and Functional Genomics, University of Florida, Gainesville, Florida, USA.
¹⁵ Santhera Pharmaceuticals, Tucson, Arizona, USA.
¹⁶ Division of Pediatric Neurology, Cincinnati Children's Hospital Medical Center, Cincinnati, Ohio, USA.

PMID: 28228973
PMCID: PMC5300692
DOI: 10.1371/currents.md.83071bbd728982f2f1073f4950e03586

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.

Grants and funding

R01 AR056973/AR/NIAMS NIH HHS/United States