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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, Lutz C, Davies K, Demonbreun 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. Gordish-Dressman H, et al. Among authors: wells dj. J Neuromuscul Dis. 2018;5(4):407-417. doi: 10.3233/JND-180324. J Neuromuscul Dis. 2018. PMID: 30198876 Free PMC article.
Translational and regulatory challenges for exon skipping therapies.
Aartsma-Rus A, Ferlini A, Goemans N, Pasmooij AM, Wells DJ, Bushby K, Vroom E, Balabanov P. Aartsma-Rus A, et al. Among authors: wells dj. Hum Gene Ther. 2014 Oct;25(10):885-92. doi: 10.1089/hum.2014.086. Hum Gene Ther. 2014. PMID: 25184444 Review.
Measuring clinical effectiveness of medicinal products for the treatment of Duchenne muscular dystrophy.
Lynn S, Aartsma-Rus A, Bushby K, Furlong P, Goemans N, De Luca A, Mayhew A, McDonald C, Mercuri E, Muntoni F, Pohlschmidt M, Verschuuren J, Voit T, Vroom E, Wells DJ, Straub V. Lynn S, et al. Among authors: wells dj. Neuromuscul Disord. 2015 Jan;25(1):96-105. doi: 10.1016/j.nmd.2014.09.003. Epub 2014 Sep 11. Neuromuscul Disord. 2015. PMID: 25307856 No abstract available.
174 results