Nancy L. Kuntz

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

Nusinersen initiated in infants during the presymptomatic stage of spinal muscular atrophy: Interim efficacy and safety results from the Phase 2 NURTURE study

Darryl C. De Vivo, Enrico Bertini, Kathryn J. Swoboda et al. · Neuromuscular Disorders · 2019 · 602 citations · Full text

Spinal Disorder, Pediatrics, Nurture Study +7

Onasemnogene abeparvovec gene therapy for symptomatic infantile-onset spinal muscular atrophy in patients with two copies of SMN2 (STR1VE): an open-label, single-arm, multicentre, phase 3 trial

John Day, Richard S. Finkel, Claudia A. Chiriboga et al. · The Lancet Neurology · 2021 · 449 citations

Rare Diseases, Spinal Cord Injury, Medicine +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