2018 Action Duchenne abstract – Longitudinal analyses of 4SC velocity in Leuven
Changes in four-stair climb (4sc) over 2 years in Duchenne muscular dystrophy (DMD): Characterizing rates of decline and prognostic factors View the Full PDF
WMS 2018 Abstract – Consistency of 6MWD
Can natural history controls be used for functional outcomes in Duchenne muscular dystrophy (DMD) drug trials? Assessing the consistency of 48-week changes in six-minute walk distance (6MWD) between multiple natural history data sources and clinical trial placebo arms. View the Full PDF
WMS 2018 Abstract – Minimal detectable change in NSAA
Minimal detectable change in the North Star Ambulatory Assessment (NSAA) in Duchenne muscular dystrophy (DMD) View the Full PDF
WMS 2018 Abstract – 4SC Velocity Prognostic Model
Prognostic factors for changes in 4-stair climb ability in patients with Duchenne muscular dystrophy (DMD) View the Full PDF
2018 Action Duchenne abstract – Consistency of 1-year change in 6MWD
Can natural history controls be used for functional outcomes in Duchenne muscular dystrophy (DMD) drug trials? Assessing the consistency of 48-week changes in six-minute walk distance (6MWD) between multiple natural history data sources and clinical trial placebo arms. View the Full PDF
Action Duchenne International Conference 2018 Abstract – Phase 1b/2 study of the anti-myostatin adnectin RG6206
Action Duchenne International Conference 2018 Abstract Nov 9th–11th, 2018, Birmingham, UK. Submission deadline: August 31st, 2018 A Phase 1b/2 study of the anti-myostatin adnectin RG6206 (BMS-986089) in ambulatory boys with Duchenne muscular dystrophy: A 72-week treatment update View the Full PDF
2018 Action Duchenne abstract – MCID of 4SC and NSAA in CCHMC
Estimating clinically meaningful change thresholds in the North Star Ambulatory Assessment (NSAA) and Four-Stair Climb (4SC) in Duchenne muscular dystrophy (DMD) View the Full PDF
2018 ICNMD – 4SC prognostic model
Development of a prognostic model for 1-year change in timed 4 Stair-Climb in Duchenne patients View the Full PDF
2017 WMS Abstract – Validation of a prognostic score for changes in 6MWD
Validation of a prognostic score for changes in six-minute walk distance (6MWD) in patients with Duchenne muscular dystrophy (DMD) View the Full PDF
2017 WMS Abstract – The Collaborative Trajectory Analysis Collaboration
The Collaborative Trajectory Analysis Collaboration (cTAP) – Accelerating the Translation of Natural History into more effective Clinical Trial Design View the Full PDF