title: Duchenne Regulatory Science Consortium Meeting on Disease Progression Modeling for Duchenne Muscular Dystrophy.
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.
authors.forename: Jane
authors.initials: J
authors.lastname: Larkindale
authors.affiliation: Department of Physical Medicine and Rehabilitation, University of California, Sacramento, California, USA.
authors.forename: Richard
authors.initials: R
authors.lastname: Abresch
authors.affiliation: Duchenne Regulatory Science Consortium, Critical Path Institute, Tucson, Arizona, USA.
authors.forename: Enrique
authors.initials: E
authors.lastname: Aviles
authors.affiliation: Parent Project Muscular Dystrophy, Hackensack, New Jersey, USA.
authors.forename: Abby
authors.initials: A
authors.lastname: Bronson
authors.affiliation: Pfizer, Rare Disease Research Unit, Cambridge, Massachusetts, USA.
authors.forename: Janice
authors.initials: J
authors.lastname: Chin
authors.affiliation: Parent Project Muscular Dystrophy, Hackensack, New Jersey, USA.
authors.forename: Pat
authors.initials: P
authors.lastname: Furlong
authors.affiliation: Children's National Medical Center, Research Center for Genetic Medicine, Washington, DC, USA.
authors.forename: Heather
authors.initials: H
authors.lastname: Gordish-Dressman
authors.affiliation: Parent Project Muscular Dystrophy, Hackensack, New Jersey, USA.
authors.forename: Elizabeth
authors.initials: E
authors.lastname: Habeeb-Louks
authors.affiliation: University of California Davis Medical Center, Department of Physical Medicine and Rehabilitation, Sacramento, California, USA.
authors.forename: Erik
authors.initials: E
authors.lastname: Henricson
authors.affiliation: PTC Therapeutics, South Plainfield, New Jersey, USA.
authors.forename: Hans
authors.initials: H
authors.lastname: Kroger
authors.affiliation: Duchenne Regulatory Science Consortium, Critical Path Institute, Tucson, Arizona, USA.
authors.forename: Charles
authors.initials: C
authors.lastname: Lynn
authors.affiliation: Stephen Lynn, Newcastle University, Newcastle upon Tyne, UK.
authors.forename: Stephen
authors.initials: S
authors.lastname: Lynn
authors.affiliation: Sarepta Therapeutics, Medical Affairs & Patient Advocacy, Cambridge, Massachusetts, USA.
authors.forename: Dana
authors.initials: D
authors.lastname: Martin
authors.affiliation: NINDS, National Institute of Health, Washington DC, USA.
authors.forename: Glen
authors.initials: G
authors.lastname: Nuckolls
authors.affiliation: Advanced Imaging Research Center, Oregon Health & Science University, Portland, Oregon, USA.
authors.forename: William
authors.initials: W
authors.lastname: Rooney
authors.affiliation: Duchenne Regulatory Science Consortium, Critical Path Institute, Tucson, Arizona, USA.
authors.forename: Klaus
authors.initials: K
authors.lastname: Romero
authors.affiliation: Department of Pharmacology and Therapeutics, University of Florida, Gainesville, Florida, USA.
authors.forename: Lee
authors.initials: L
authors.lastname: Sweeney
authors.affiliation: Department of Physical Therapy, University of Florida, Gainesville, Florida, USA.
authors.forename: Krista
authors.initials: K
authors.lastname: Vandenborne
authors.affiliation: Department of Physiology and Functional Genomics, University of Florida, Gainesville, Florida, USA.
authors.forename: Glenn
authors.initials: G
authors.lastname: Walter
authors.affiliation: Santhera Pharmaceuticals, Tucson, Arizona, USA.
authors.forename: Jodi
authors.initials: J
authors.lastname: Wolff
authors.affiliation: Division of Pediatric Neurology, Cincinnati Children's Hospital Medical Center, Cincinnati, Ohio, USA.
authors.forename: Brenda
authors.initials: B
authors.lastname: Wong
authors.affiliation: Department of Physical Medicine and Rehabilitation, University of California, Sacramento, California, USA.
authors.forename: Craig M
authors.initials: CM
authors.lastname: McDonald
authors.forename: Members Of The
authors.initials: MO
authors.lastname: Duchenne Regulatory Science Consortium Imaging-Dmd Consortium And The Cinrg Investigators