Quantitative magnetic resonance imaging measures as biomarkers of disease progression in boys with Duchenne muscular dystrophy: a phase 2 trial of domagrozumab.
Sherlock, SP; Palmer, J; Wagner, KR; Abdel-Hamid, HZ; Bertini, E; Tian, C; Mah, JK; Kostera-Pruszczyk, A; Muntoni, F; Guglieri, M; et al.
Journal of Neurology.
2022.
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.
Muntoni, F; Signorovitch, J; Sajeev, G; Goemans, N; Wong, B; Tian, C; Mercuri, E; Done, N; Wong, H; Moss, J; et al.
Neuromuscular Disorders.
2022;
32:271-283.
Repeated intravenous cardiosphere-derived cell therapy in late-stage Duchenne muscular dystrophy (HOPE-2): a multicentre, randomised, double-blind, placebo-controlled, phase 2 trial.
McDonald, CM; Marbán, E; Hendrix, S; Hogan, N; Ruckdeschel Smith, R; Eagle, M; Finkel, RS; Tian, C; Janas, J; Harmelink, MM; et al.
The Lancet.
2022;
399:1049-1058.
The effect of oral bisphosphonate therapy on vertebral morphometry and fractures in patients with Duchenne muscular dystrophy and glucocorticoid-induced osteoporosis.
Nasomyont, N; Tian, C; Hornung, L; Khoury, J; Hochwalt, PM; Tilden, JC; Wong, BL; Rutter, MM.
Muscle and Nerve.
2021;
64:710-716.
Emergency Planning as Part of Healthcare Transition Preparation for Patients with Duchenne Muscular Dystrophy.
Chouteau, WA; Burrows, C; Wittekind, SG; Rutter, MM; Bange, JE; Sabla, GE; Rybalsky, I; Tian, C.
Journal of Pediatric Nursing.
2021;
61:298-304.
The Effect of Adiposity on Cardiovascular Function and Myocardial Fibrosis in Patients With Duchenne Muscular Dystrophy.
Henson, SE; Lang, SM; Khoury, PR; Tian, C; Rutter, MM; Urbina, EM; Ryan, TD; Taylor, MD; Alsaied, T.
Journal of the American Heart Association.
2021;
10.
DMD – CLINICAL CARE EP.101 Appendicular lean mass index (ALMI) changes in patients with dystrophinopathies.
Wong, B; Summer, S; Horn, P; Rutter, M; Tian, C; Rybalsky, I; Shellenbarger, K; Kalkwarf, H.
Neuromuscular Disorders.
2021;
31.
DMD - TREATMENT EP.143 Assessment of patients on Eteplirsen (Exondys 51) therapy using real world data.
Zygmunt, A; Rybalsky, I; Chouteau, W; Horn, P; Tian, C.
Neuromuscular Disorders.
2021;
31.
DMD/BMD – OUTCOME MEASURES EP.125 Minimal detectable changes in functional measures in Duchenne muscular dystrophy (DMD): A study of multiple centers, networks and trial arms.
Muntoni, F; Signorovitch, J; Sajeev, G; Done, N; Yao, Z; Goemans, N; McDonald, C; Mercuri, E; Niks, E; Wong, B; et al.
Neuromuscular Disorders.
2021;
31.
DMD/BMD – OUTCOME MEASURES EP.122 Prognostic factors for pulmonary milestones in Duchenne muscular dystrophy (DMD).
Goemans, N; Signorovitch, J; Sajeev, G; Wong, B; Tian, C; McDonald, C; Mercuri, E; Niks, E; Freimark, J; Jenkins, M; et al.
Neuromuscular Disorders.
2021;
31.