Challenges of clinical trial design for DMD
Ricotti V, Muntoni F, Voit T. Challenges of clinical trial design for DMD. Neuromuscular disorders : NMD 2015;25:932-5 Including a…
The effect of steroid treatment on weight in nonambulatory males with Duchenne muscular dystrophy
Lamb MM, Cai B, Royer J, et al. The effect of steroid treatment on weight in nonambulatory males with Duchenne…
Neurodevelopmental Needs in Young Boys with Duchenne Muscular Dystrophy (DMD): Observations from the Cooperative International Neuromuscular Research Group (CINRG) DMD Natural History Study (DNHS)
Thangarajh M, Spurney CF, Gordish-Dressman H, et al. Neurodevelopmental Needs in Young Boys with Duchenne Muscular Dystrophy (DMD): Observations from…
Longitudinal pulmonary function testing outcome measures in Duchenne muscular dystrophy: Long-term natural history with and without glucocorticoids
McDonald CM, Gordish-Dressman H, Henricson EK, et al. Longitudinal pulmonary function testing outcome measures in Duchenne muscular dystrophy: Long-term natural…
Fracture in Duchenne Muscular Dystrophy: Natural History and Vitamin D Deficiency
Perera N, Sampaio H, Woodhead H, Farrar M. Fracture in Duchenne Muscular Dystrophy: Natural History and Vitamin D Deficiency. Journal…
The NorthStar Ambulatory Assessment in Duchenne muscular dystrophy: considerations for the design of clinical trials
Ricotti V, Ridout DA, Pane M, et al. The NorthStar Ambulatory Assessment in Duchenne muscular dystrophy: considerations for the design…
What can Duchenne Connect teach us about treating Duchenne muscular dystrophy?
Wang RT, Nelson SF. What can Duchenne Connect teach us about treating Duchenne muscular dystrophy? Current opinion in neurology 2015;28:535-41.…
Prednisone/prednisolone and deflazacort regimens in the CINRG Duchenne Natural History Study
Bello L, Gordish-Dressman H, Morgenroth LP, et al. Prednisone/prednisolone and deflazacort regimens in the CINRG Duchenne Natural History Study. Neurology…
Characterization of Pulmonary Function in Duchenne Muscular Dystrophy
Mayer OH, Finkel RS, Rummey C, et al. Characterization of pulmonary function in Duchenne Muscular Dystrophy. Pediatric pulmonology 2015;50:487-94. Commentary…
Genetic Modifiers of Ambulation in the Cooperative International Neuromuscular Research Group Duchenne Natural History Study
Bello L, Kesari A, Gordish-Dressman H, et al. Genetic modifiers of ambulation in the Cooperative International Neuromuscular Research Group Duchenne…
