MR biomarkers predict clinical function in Duchenne muscular dystrophy
Alison M. Barnard, Rebecca J. Willcocks, William Triplett, Sean C. Forbes, Michael J. Daniels, Saptarshi Chakraborty, Donovan J. Lott, Claudia Senesac, Erika Finanger, Ann T. Harrington, Gihan Tennekoon, Harneet Arora, Dah-Jyuu Wang, H. Lee Sweeney, William D. Rooney, Glenn A. Walter, Krista Vandenborne
Abstract
OBJECTIVE: To investigate the potential of lower extremity magnetic resonance (MR) biomarkers to serve as endpoints in clinical trials of therapeutics for Duchenne muscular dystrophy (DMD) by characterizing the longitudinal progression of MR biomarkers over 48 months and assessing their relationship to changes in ambulatory clinical function. METHODS: biomarkers of disease progression. In addition, 4 tests of ambulatory function were performed. Participants returned for follow-up data collection at 12, 24, 36, and 48 months. RESULTS: were the strongest predictors of future loss of function, including loss of ambulation. CONCLUSIONS: This study supports the strong relationship between lower extremity MR biomarkers and measures of clinical function, as well as the ability of MR biomarkers, particularly those from proximal muscles, to predict future ambulatory function and important clinical milestones. CLINICALTRIALSGOV IDENTIFIER: NCT01484678.