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;
269:4421-4435.
Obesity and loss of ambulation are associated with lower extremity oedema in Duchenne muscular dystrophy.
Freytag, JM; Ryan, TD; Bange, JE; Bonarrigo, KC; Chouteau, WA; Wittekind, SG; Tian, C; Gao, Z; Villa, CR.
Cardiology in the Young.
2022;
1-6.
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.
A Randomized, Double-Blind, Placebo-Controlled, Global Phase 3 Study of Edasalonexent in Pediatric Patients with Duchenne Muscular Dystrophy: Results of the PolarisDMD Trial.
Finkel, RS; McDonald, CM; Lee Sweeney, H; Finanger, E; Neil Knierbein, E; Wagner, KR; Mathews, KD; Marks, W; Statland, J; Nance, J; et al.
Journal of Neuromuscular Diseases.
2021;
8:769-784.
Treatment of SCN4A-induced myotonic crisis.
Ritter, DM; Tian, C; Broomall, E.
Muscle and Nerve.
2021;
63:E59-E61.
An autosomal dominant neurological disorder caused by de novo variants in FAR1 resulting in uncontrolled synthesis of ether lipids.
Ferdinandusse, S; McWalter, K; te Brinke, H; IJlst, L; Mooijer, PM; Ruiter, JP N; van Lint, AE M; Pras-Raves, M; Wever, E; Millan, F; et al.
Genetics in Medicine.
2021;
23:740-750.