Results 181 to 190 of about 2,876 (205)

Treatment of infantile-onset spinal muscular atrophy with nusinersen: a phase 2, open-label, dose-escalation study

Lancet, The, 2016
Richard S Finkel, Claudia A Chiriboga, John W Day   +2 more
exaly  

Spinal muscular atrophy

Lancet, The, 2008
Mitchell R Lunn
exaly  

Exon skipping and dystrophin restoration in patients with Duchenne muscular dystrophy after systemic phosphorodiamidate morpholino oligomer treatment: an open-label, phase 2, dose-escalation study

Lancet, The, 2011
Sebahattin Cirak, V Arechavala-Gomeza, Karen Anthony   +2 more
exaly  

Long-term effects of glucocorticoids on function, quality of life, and survival in patients with Duchenne muscular dystrophy: a prospective cohort study

Lancet, The, 2018
Craig M Mcdonald, Erik K Henricson, Paula R Clemens   +2 more
exaly  

Genetic Predictors and Remodeling of Dilated Cardiomyopathy in Muscular Dystrophy

Circulation, 2005
John L Jefferies, John W Belmont, JEFFREY TOWBIN   +2 more
exaly  

Nusinersen versus Sham Control in Infantile-Onset Spinal Muscular Atrophy

New England Journal of Medicine, 2017
Richard S Finkel, Eugenio Mercuri, Basil T Darras   +2 more
exaly  

Single-Dose Gene-Replacement Therapy for Spinal Muscular Atrophy

New England Journal of Medicine, 2017
Richard Shell, Linda P Lowes, Lindsay N Alfano   +2 more
exaly  

Efficacy of idebenone on respiratory function in patients with Duchenne muscular dystrophy not using glucocorticoids (DELOS): a double-blind randomised placebo-controlled phase 3 trial

Lancet, The, 2015
Maria Grazia D'Angelo, Richard S Finkel, Craig M Mcdonald   +2 more
exaly  

Dystrophin Immunity in Duchenne's Muscular Dystrophy

New England Journal of Medicine, 2010
Louise Rodino-Klapac, Zarife Sahenk, Dawn Bowles   +2 more
exaly  

Nusinersen versus Sham Control in Later-Onset Spinal Muscular Atrophy

New England Journal of Medicine, 2018
Eugenio Mercuri, Basil T Darras, Claudia A Chiriboga   +2 more
exaly