Results 51 to 60 of about 1,629 (69)

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  

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
Tina Duong, Eric P Hoffman, Heather Gordish-Dressman   +2 more
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, Michela Guglieri   +2 more
exaly  

Signs and symptoms of Duchenne muscular dystrophy and Becker muscular dystrophy among carriers in the Netherlands: a cohort study

Lancet, The, 1999
Egbert Bakker
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
Thomas Voit, Ulrike schara, Maria Grazia D'Angelo   +2 more
exaly  

Spinal muscular atrophy

Lancet, The, 2008
Mitchell R Lunn
exaly  

Muscular dystrophies

Lancet, The, 2019
Eugenio Mercuri, Carsten G Bönnemann, Francesco Muntoni   +2 more
exaly  

Molecular mechanisms of muscular dystrophies: old and new players

Nature Reviews Molecular Cell Biology, 2006
Kay E Davies, Kristen J Nowak
exaly  

Randomised trial of preventive nasal ventilation in Duchenne muscular dystrophy

Lancet, The, 1994
Sylvie Chevret
exaly  

Location of facioscapulohumeral muscular dystrophy gene on chromosome 4

Lancet, The, 1990
Cisca Wijmenga
exaly