{"id":"W3174155996","doi":"10.1002/mus.27363","title":"Expert recommendations and clinical considerations in the use of onasemnogene abeparvovec gene therapy for spinal muscular atrophy","year":2021,"lang":"en","type":"review","venue":"Muscle & Nerve","topic":"Neurogenetic and Muscular Disorders Research","field":"Medicine","cited_by":66,"is_retracted":false,"has_abstract":true,"ca_institutions":"Children's Hospital of Eastern Ontario; University of Ottawa","funders":"","keywords":"Spinal muscular atrophy; SMA*; Medicine; SMN1; Disease; Neuromuscular disease; Intensive care medicine; Genetic enhancement; Pediatrics; Physical therapy; Bioinformatics; Physical medicine and rehabilitation; Internal medicine","routes":{"ca_aff":true,"ca_fund":false,"ca_venue":false,"about_ca":false,"invisible_to_affiliation_only":false},"retraction":null,"screen":null,"direct_labels":[],"prediction":{"model_version":"metacan-v3-hybrid-931329e0061c","candidate_categories":[],"consensus_categories":[],"category_scores_codex":[0.009671157,0.0007961873,0.001452959,0.0009317092,0.001316542,0.003141504,0.003138553,0.01519981,0.02208909],"category_scores_gemma":[0.06008905,0.0004291299,0.002236697,0.0006027518,0.00148587,0.003357073,0.001776099,0.009858384,0.01491624],"about_ca_system_candidate":false,"about_ca_system_consensus":false,"about_ca_system_score_codex":0.002022355,"about_ca_system_score_gemma":0.005105117,"about_ca_topic_candidate":false,"about_ca_topic_consensus":false,"about_ca_topic_score_codex":0.003111698,"about_ca_topic_score_gemma":0.007993646,"domain_scores_codex":[0.9900343,0.00521488,0.001430699,0.000487366,0.002294931,0.0005379443],"domain_scores_gemma":[0.9592069,0.01847364,0.002287839,0.0007347704,0.01646107,0.002835743],"domain_codex":null,"domain_gemma":null,"domain_candidate":null,"domain_consensus":null,"study_design_codex":"not_applicable","study_design_gemma":"not_applicable","study_design_scores_codex":[0.00008344576,0.00007648392,0.0003554247,0.001793671,0.00004451302,0.0006945294,0.0002294937,0.0002581256,0.0002390247,0.00216926,0.8262507,0.1678053],"study_design_scores_gemma":[0.0001454231,0.00007238286,0.0004658624,0.008880475,0.00009984853,0.001131884,0.0004242767,0.0002398005,0.0001907498,0.006896033,0.9813887,0.00006459974],"study_design_candidate":"not_applicable","study_design_consensus":"not_applicable","genre_codex":"commentary","genre_gemma":"review","genre_scores_codex":[0.001201636,0.07390272,0.005579265,0.839058,0.02175201,0.0007073177,0.0007105609,0.0004418908,0.05664665],"genre_scores_gemma":[0.0216695,0.1040754,0.04131292,0.753388,0.03146443,0.002886293,0.001505871,0.0002442067,0.0434534],"genre_candidate":"review","genre_consensus":null,"teacher_disagreement_score":0.02208909,"threshold_uncertainty_score":0.07389539,"prediction_status":"machine_predicted_unvalidated"},"machine_scores":{"provisional":true,"baseline":true,"maturity_gate_passed":false,"score_opus":0.3850230911027996,"score_gpt":0.4869923627829738,"score_spread":0.1019692716801742,"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."}}