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Scientific Articles

“Of Mice and Measures”: A Project to Improve How We Advance Duchenne Muscular Dystrophy Therapies to the Clinic First Workshop Report: Examining current findings and opportunities around the emerging D2.B10-Dmdmdx/J (D2/mdx) model in context of the classic C57BL/10ScSn-Dmdmdx/J (Bl10/mdx)

key information

source: Journal of Neuromuscular Diseases

year: 2018

authors: Gordish-Dressman H, Willmann R, Dalle Pazze L, Kreibich A, van Putten M, Heydemann A, Bogdanik L, Lutz C, Davies K, Demonbruen AR, Duan D, Elsey D, Fukada SI, Girgenrath M, Patrick Gonzalez J, Grounds MD, Nichols A, Partridge T, Passini M, Sanarica F, Schnell FJ, Wells DJ, Yokota T, Young CS, Zhong Z, Spurney C, Spencer M, De Luca A, Nagaraju K, Aartsma-Rus A


A new line of dystrophic mdx mice on the DBA/2J (D2) background has emerged as a candidate to study the efficacy of therapeutic approaches for Duchenne muscular dystrophy (DMD). These mice harbor genetic polymorphisms that appear to increase the severity of the dystropathology, with disease modifiers that also occur in DMD patients, making them attractive for efficacy studies and drug development. This workshop aimed at collecting and consolidating available data on the pathological features and the natural history of these new D2/mdx mice, for comparison with classic mdx mice and controls, and to identify gaps in information and their potential value. The overall aim is to establish guidance on how to best use the D2/mdx mouse model in preclinical studies.

organization: Children's National Medical Center, USA; University of Basel, Switzerland; Charley's Fund, USA; Leiden University Medical Center, Netherlands; University of Illinois at Chicago, USA; The Jackson Laboratory, USA; University of Oxford, UK; Northwestern University Feinberg School of Medicine, USA; University of Missouri, USA; Summit Therapeutics, UK; Osaka University, Japan; Pfizer, USA; Solid Biosciences, USA; University of Western Australia, Australia; Catabasis Pharmaceuticals, USA; Sarepta Therapeutics Inc., USA; University of Bari "Aldo Moro", Italy; Royal Veterinary College, UK; University of Alberta, Canada; Center for Duchenne Muscular Dystrophy at UCLA, USA; Wave Life Sciences, USA; Binghamton University, USA

DOI: 10.3233/JND-180324

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