Categorising trajectories and individual item changes of the North Star Ambulatory Assessment in patients with Duchenne muscular dystrophy
PLOS One, September 3, 2019
The scientific journal PLOS One has published the results of a research study using real-world data from patients with Duchenne muscular dystrophy (DMD), a rare progressive muscle-weakening and wasting disease. The study team characterized the progression of the disease based on a range of functional outcomes that reflect DMD patients’ ambulatory function. The study was conducted as part of the Collaborative Trajectory Analysis Project (cTAP), a coalition of drug developers, academic experts, and patient advocates focused on learning from patient data to improve drug development in DMD. The research was conducted in collaboration with the UK North Star Clinical Network by a data science team led by Analysis Group Managing Principal James Signorovitch and including Vice President Gautam Sajeev, who also coauthored the paper.
The article, “Categorising trajectories and individual item changes of the North Star Ambulatory Assessment in patients with Duchenne muscular dystrophy,” reports on the results of a study of 395 patients with DMD. The findings provide quantitative insights that can be used to assist academics, drug developers, and regulators in clinical trial design and analysis.
Authors
Muntoni F, Domingos J, Manzur AY, Mayhew A, Guglieri M, The UK NorthStar Network, Sajeev G, Signorovitch J, Ward SJ