Brenda Wong

Ataluren in patients with nonsense mutation Duchenne muscular dystrophy (ACT DMD): a multicentre, randomised, double-blind, placebo-controlled, phase 3 trial

R. Finkel, E. Bertini, S. Peltz, 2017, The Lancet.

Real-world and natural history data for drug evaluation in Duchenne muscular dystrophy: suitability of the North Star Ambulatory Assessment for comparisons with external controls

Hallee E. Wong, E. Mercuri, F. Muntoni, 2022, Neuromuscular Disorders.

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 collaboration

N. Goemans, M. Van den Hauwe, D. Cox, 2020, PloS one.

Duchenne Regulatory Science Consortium Meeting on Disease Progression Modeling for Duchenne Muscular Dystrophy

R. Abresch, W. Rooney, K. Romero, 2017, PLoS currents.