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"Of Mice and Measures": A Project to Improve How We Advance Duchenne Muscular Dystrophy Therapies to the Clinic

Gordish-Dressman, H and Willmann, R and Dalle Pazze, L and Kreibich, A and van Putten, M and Heydemann, A and Bogdanik, L and Davies, K and Demonbruen, A and Duan, D and Elsey, D and Fukada, S and Girgenrath, S and Gonzalez, P and Grounds, M and Nichols, A and Partridge, T and Passini, M and Sanarica, F and Schnell, F and Wells, D J and Yokota, T and Young, C and Zhong, Z and Spurney, C and Spencer, M and De Luca, A and Nagaraju, K and Aartsma-Rus, A (2018) "Of Mice and Measures": A Project to Improve How We Advance Duchenne Muscular Dystrophy Therapies to the Clinic. Journal of Neuromuscular Diseases, 5 (4). pp. 407-414.

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Abstract

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.

Item Type: Article
RVC Publication Type: Research (full) paper
DOI: https://doi.org/10.3233/JND-180324
Departments: Comparative Biomedical Sciences
Research Programmes: Comparative Physiology & Medicine > Musculoskeletal Biology
Depositing User: RVC Auto-import
Date Deposited: 03 Nov 2018 06:00
Last Modified: 07 Nov 2018 12:06
URI: http://researchonline.rvc.ac.uk/id/eprint/11793