Finkel RS, Mercuri E, Darras BT, et al. Nusinersen versus sham control in infantile-onset spinal muscular atrophy. N Engl J Med 2017; 377: 1723–32.
Aragon-Gawinska K, Seferian AM, Daron A, et al. Nusinersen in spinal muscular atrophy type 1 patients older than 7 months: a cohort study. Neurology 2018; 91: e1312–8.
Gidaro T, Servais L. Nusinersen treatment of spinal muscular atrophy: current knowledge and existing gaps. Dev Med Child Neurol 2019; 61: 19–24.
Alfano L, Lowes L, Berry K, Flanigan K, Cripe L, Mendell J. Role of motivation on performance of the 6-minute walk test in boys with Duchenne muscular dystrophy. Dev Med Child Neurol 2015; 57(Suppl. 5): S57–8.
Alfano LN, Miller NF, Iammarino MA, et al. Ability Captured Through Interactive Video Evaluation workspace volume video game to quantify meaningful change in spinal muscular atrophy. Dev Med Child Neurol 2020; 62: 303–9.