M. Tulinius

Nusinersen versus Sham Control in Infantile-Onset Spinal Muscular Atrophy

Richard S. Finkel, Eugenio Mercuri, Basil T. Darras et al. · New England Journal of Medicine · 2017 · 2.2K citations · Full text

Nusinersen versus Sham Control in Later-Onset Spinal Muscular Atrophy

Eugenio Mercuri, Basil T. Darras, Claudia A. Chiriboga et al. · New England Journal of Medicine · 2018 · 1.4K citations

Systemic Administration of PRO051 in Duchenne's Muscular Dystrophy

Nathalie Goemans, M. Tulinius, Johanna T. van den Akker et al. · New England Journal of Medicine · 2011 · 659 citations · Full text

Ataluren in patients with nonsense mutation Duchenne muscular dystrophy (ACT DMD): a multicentre, randomised, double-blind, placebo-controlled, phase 3 trial

Craig M. McDonald, Craig Campbell, Ricardo Erazo Torricelli et al. · The Lancet · 2017 · 446 citations · Full text

Rare Diseases, Urology, Clinical Trials +5

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

Craig M. McDonald, Erik Henricson, Richard T. Abresch et al. · The Lancet · 2017 · 446 citations

Long-term Effects, Duchenne Muscular Dystrophy, Degenerative Disease +4