{"id":"W2965743431","doi":"10.1136/jnnp-2019-anzan.14","title":"014 AVXS-101 gene-replacement therapy (GRT) in presymptomatic spinal muscular atrophy (SMA): study update","year":2019,"lang":"en","type":"article","venue":"Journal of Neurology Neurosurgery & Psychiatry","topic":"Neurogenetic and Muscular Disorders Research","field":"Medicine","cited_by":3,"is_retracted":false,"has_abstract":true,"ca_institutions":"Children's Hospital of Eastern Ontario","funders":"","keywords":"SMA*; Spinal muscular atrophy; SMN1; Medicine; Asymptomatic; Pediatrics; Internal medicine; Neuromuscular disease; Disease","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":[0.001993233,0.0006255637,0.001867876,0.0009085486,0.0002029003,0.001382514,0.00110429,0.001200547,0.004562268],"category_scores_gemma":[0.002677663,0.0002192804,0.001416248,0.001117761,0.0003574333,0.0009013918,0.0003703493,0.002008952,0.001087325],"about_ca_system_candidate":false,"about_ca_system_consensus":false,"about_ca_system_score_codex":0.0006306595,"about_ca_system_score_gemma":0.001073362,"about_ca_topic_candidate":false,"about_ca_topic_consensus":false,"about_ca_topic_score_codex":0.001280677,"about_ca_topic_score_gemma":0.004408493,"domain_scores_codex":[0.9996774,0.0001217849,0.00006348278,0.00004348295,0.00006450745,0.00002928866],"domain_scores_gemma":[0.9977294,0.001347435,0.0002941174,0.0000467587,0.0004400135,0.0001423072],"domain_codex":null,"domain_gemma":null,"domain_candidate":null,"domain_consensus":null,"study_design_codex":"design_other","study_design_gemma":"nonrandomized_trial","study_design_scores_codex":[0.003128416,0.0004652761,0.001491363,0.01703173,0.001258579,0.0001806691,0.00008585698,0.000371612,0.001799922,0.001432685,0.03913749,0.9336164],"study_design_scores_gemma":[0.007436804,0.0134875,0.01409973,0.04143136,0.01009472,0.00202957,0.000279951,0.0009221324,0.002111156,0.003078957,0.9049327,0.00009538233],"study_design_candidate":"nonrandomized_trial","study_design_consensus":null,"genre_codex":"review","genre_gemma":"empirical","genre_scores_codex":[0.005299138,0.985198,0.0003785722,0.003149387,0.0007911573,0.0001245447,0.0004148267,0.00004419792,0.004600338],"genre_scores_gemma":[0.02651874,0.9605013,0.001305438,0.005652119,0.002270792,0.0003245307,0.001138922,0.0000300325,0.002258262],"genre_candidate":"empirical","genre_consensus":null,"teacher_disagreement_score":0.004562268,"threshold_uncertainty_score":0.01526225,"prediction_status":"machine_predicted_unvalidated"},"machine_scores":{"provisional":true,"baseline":true,"maturity_gate_passed":false,"score_opus":0.01332983667601602,"score_gpt":0.2923654605917728,"score_spread":0.2790356239157568,"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."}}