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
cTAP to Present Late-breaking Results at the World Muscle Society Congress
Published October 1, 2018 The collaborative Trajectory Analysis Project (cTAP), a multi-stakeholder, non-competitive global coalition in Duchenne muscular dystrophy, today announced the acceptance of a late-breaking abstract for presentation at the 23rd International Congress of the World Muscle Society. This is in addition to the two previously accepted submissions that cTAP collaborators will present at the Congress, which is being held October 2-6, 2018 at the Intercontinental Hotel in Mendoza, Argentina. READ FULL ARTICLE: cTAP...
cTAP Presentation at the 2018 ASA Biopharmaceutical Section Regulatory-Industry Statistics Workshop
Susan J. Ward presented at the ASA Biopharmaceutical Section Regulatory-Industry Statistics Workshop Annual Conference 2018. The presentation was entitled “Collaborative Learning from Patient Data in Rare Disease”. SEE FULL PRESENTATION
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