2019 JSCN Abstract – Phase 1b/2 Study of RG6206
A Phase 1b/2 study of RG6206 (BMS-986089) in boys with Duchenne muscular dystrophy: 72-week update View the Full PDF
2019 MDA Abstract – NSAA Shift Analyses
One-year shifts in North Star Ambulatory Assessment items in Duchene muscular dystrophy (DMD) patients in the UK NorthStar Network database View the Full PDF
2019 MDA Abstract – The Collaborative Trajectory Analysis Project (cTAP)
The collaborative Trajectory Analysis Project (cTAP) – pioneering collaborative analytics for more effective clinical trials in Duchenne Muscular Dystrophy View the Full PDF
2019 PTC WMS Abstract – Steroid Use and DMD
DEFLAZACORT OR PREDNISONE TREATMENT FOR DUCHENNE MUSCULAR DYSTROPHY (DMD): REAL-WORLD OUTCOMES AT CINCINNATI CHILDREN’S HOSPITAL MEDICAL CENTER (CCHMC) View the Full PDF
2019 WMS Abstract – Prognostic score for loss of walking ability in DMD
A composite prognostic score for time to loss of walking ability in Duchenne muscular dystrophy (DMD) View the Full PDF
2019 WMS Abstract – 48-Week Changes in NSAA
Consistency of 48-week changes in North Star Ambulatory Assessment (NSAA) between Duchenne muscular dystrophy (DMD) natural history data and clinical trial placebo arms, after adjustment for prognostic factors View the Full PDF
2019 WMS Abstract – Lean Body Mass and Motor Function in DMD Patients
REAL-WORLD ASSOCIATIONS BETWEEN MOTOR FUNCTION AND LEAN BODY MASS IN THE ARMS AND LEGS IN PATIENTS WITH DUCHENNE MUSCULAR DYSTROPHY View the Full PDF
2019 WMS Abstract – Estimating Changes in NSAA and 4SC in DMD
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
2019 Myology Abstract – Changes in 4SC over Two Years in DMD
Changes in Four-Stair Climb (4SC) over Two Years in Duchenne Muscular Dystrophy (DMD): Consistency across Data Sources and Prognostic Factors View the Full PDF
2018 ISPOR EU Abstract – Ambulatory Function in Duchenne Muscular Dystrophy
Ambulatory function in Duchenne muscular dystrophy (DMD): The characteristic trajectory and variation across individuals View the Full PDF