News & Events
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January 8, 2021
Effective Data Sharing as a Conduit for Advancing Medical Product Development
Karpen SR, White JK, Mullin AP, O’Doherty I, Hudson LD, Romero K, Sivakumaran S, Stephenson D, Turner EC, Larkindale J. Effective Data Sharing as a Conduit for Advancing Medical Product Development. Ther Innov Regul Sci. 2021 May;55(3):591-600... -
June 14, 2020
Standardized Data Structures in Rare Diseases: CDISC User Guides for Duchenne Muscular Dystrophy and Huntington’s Disease
Mullin, A.P., Corey, D., Turner, E.C., Liwski, R., Olson, D., Burton, J., Sivakumaran, S., Hudson, L.D., Romero, K., Stephenson, D.T. and Larkindale, J. (2021), Standardized Data Structures in Rare Diseases: CDISC User Guides for Duchenne Muscular Dystrophy and Huntington’s Disease. Clin Transl Sci, 14: 214-221.... - May 14, 2020
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August 14, 2019
Conrado et al. 2019 Towards Regulatory Endorsement
Towards regulatory endorsement of drug development tools to promote the application of model-informed drug development in Duchenne muscular dystrophy.... -
October 26, 2017
226th ENMC International Workshop: Towards Validated and Qualified Biomarkers for Therapy Development for Duchenne Muscular Dystrophy
Aartsma-Rus A, Ferlini A, McNally EM, Spitali P, Sweeney HL, Aartsma-Rus AM, Szigyarto CAK, Bello L, Bronson A, Brown K, Buccella F, Chadwick J, Ferlini A, Frank D, Hoffman E, Larkindale J, McClorey G, McNally E, Munschauer R, Muntoni F, Owens J, Schara U, Spitali P, Straub V, Sweeney L, Tinsley J, Versnel J, Vroom E, Welch E. 226th ENMC International Workshop: Towards Validated and Qualified Biomarkers for Therapy Development for Duchenne Muscular Dystrophy. Neuromuscular Disorders. 2017 Oct.