{"id":"W2560202024","doi":"10.1016/s0140-6736(16)31408-8","title":"Treatment of infantile-onset spinal muscular atrophy with nusinersen: a phase 2, open-label, dose-escalation study","year":2016,"lang":"en","type":"article","venue":"The Lancet","topic":"Neurogenetic and Muscular Disorders Research","field":"Medicine","cited_by":1016,"is_retracted":false,"has_abstract":false,"ca_institutions":"Hospital for Sick Children; University of Toronto","funders":"","keywords":"Medicine; Spinal muscular atrophy; SMN1; Tolerability; Adverse effect; Motor neuron; Atrophy; Pediatrics; Internal medicine; Surgery; Disease","routes":{"ca_aff":true,"ca_fund":false,"ca_venue":false,"about_ca":false,"invisible_to_affiliation_only":false},"retraction":null,"screen":null,"machine_scores":{"provisional":true,"baseline":true,"maturity_gate_passed":false,"score_opus":0.08320113110198354,"score_gpt":0.3974570351688934,"score_spread":0.3142559040669098,"validation_status":"score_only:v0-immature-baseline","note":"Baseline scores from an immature model (maturity gate not passed). Scores rank; they never assert a category."}}