Categorising trajectories and individual item changes of the North Star Ambulatory Assessment in patients with Duchenne muscular dystrophy.

by Francesco Muntoni, Joana Domingos, Adnan Y Manzur, Anna Mayhew, Michela Guglieri, UK NorthStar Network, Gautam Sajeev, James Signorovitch, Susan J Ward
Published 2019-01-01

Primary Hyperoxaluria Type 1 Disease Manifestations and Healthcare Utilization: A Multi-Country, Online, Chart Review Study

by Xiangling Wang, David Danese, Thomas Brown, Jessica Baldwin, Gautam Sajeev, Erin E. Cook, Yao Wang, Chunyi Xu, Hongbo Yang, Michael L. Moritz
Published 2021-09-01

Prognostic factors for changes in the timed 4-stair climb in patients with Duchenne muscular dystrophy, and implications for measuring drug efficacy: A multi-institutional collabor...

by Nathalie Goemans, Brenda Wong, Marleen Van den Hauwe, James Signorovitch, Gautam Sajeev, David Cox, John Landry, Madeline Jenkins, Ibrahima Dieye, Zhiwen Yao, Intekhab Hossain, Susan J Ward, Collaborative Trajectory Analysis Project (cTAP)
Published 2020-01-01