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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; Willmann, R; Dalle Pazze, L; Kreibich, A; van Putten, M; Heydemann, A; Bogdanik, L; Davies, K; Demonbruen, A; Duan, D; Elsey, D; Fukada, S; Girgenrath, S; Gonzalez, P; Grounds, M; Nichols, A; Partridge, T; Passini, M; Sanarica, F; Schnell, F; Wells, D J; Yokota, T; Young, C; Zhong, Z; Spurney, C; Spencer, M; De Luca, A; Nagaraju, K; Aartsma-Rus, A

Authors

H Gordish-Dressman

R Willmann

L Dalle Pazze

A Kreibich

M van Putten

A Heydemann

L Bogdanik

K Davies

A Demonbruen

D Duan

D Elsey

S Fukada

S Girgenrath

P Gonzalez

M Grounds

A Nichols

T Partridge

M Passini

F Sanarica

F Schnell

D J Wells

T Yokota

C Young

Z Zhong

C Spurney

M Spencer

A De Luca

K Nagaraju

A Aartsma-Rus



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.

Citation

Gordish-Dressman, H., Willmann, R., Dalle Pazze, L., Kreibich, A., van Putten, M., Heydemann, A., …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), 407-414. https://doi.org/10.3233/JND-180324

Journal Article Type Article
Acceptance Date Oct 1, 2018
Publication Date Oct 23, 2018
Deposit Date Nov 3, 2018
Publicly Available Date Mar 28, 2024
Journal Journal of Neuromuscular Diseases
Print ISSN 2214-3599
Publisher IOS Press
Peer Reviewed Peer Reviewed
Volume 5
Issue 4
Pages 407-414
DOI https://doi.org/10.3233/JND-180324
Public URL https://rvc-repository.worktribe.com/output/1385468