{"id":"W4247792675","doi":"10.1017/cjn.2019.165","title":"P.065 AVXS-101 gene-replacement therapy (GRT)) in presymptomatic spinal muscular atrophy (SMA): study update","year":2019,"lang":"en","type":"article","venue":"Canadian Journal of Neurological Sciences / Journal Canadien des Sciences Neurologiques","topic":"Neurogenetic and Muscular Disorders Research","field":"Medicine","cited_by":1,"is_retracted":false,"has_abstract":true,"ca_institutions":"","funders":"","keywords":"SMA*; Medicine; SMN1; Spinal muscular atrophy; Asymptomatic; Internal medicine; Gastroenterology; Disease","routes":{"ca_aff":false,"ca_fund":false,"ca_venue":true,"about_ca":false,"invisible_to_affiliation_only":true},"retraction":null,"screen":null,"direct_labels":[],"prediction":{"model_version":"codex-gemma-dda1882f352a","candidate_categories":["metaepi_narrow","sts"],"consensus_categories":[],"category_scores_codex":[0.005083521,0.0004189736,0.0007974672,0.00154358,0.001067103,0.0004935865,0.001826223,0.0001653314,0.000576321],"category_scores_gemma":[0.0007756047,0.0002758972,0.0003635632,0.001756845,0.002807556,0.0005543383,0.0001078281,0.001492316,0.00002173998],"about_ca_system_candidate":false,"about_ca_system_consensus":false,"about_ca_system_score_codex":0.0002584709,"about_ca_system_score_gemma":0.003874739,"about_ca_topic_candidate":false,"about_ca_topic_consensus":false,"about_ca_topic_score_codex":0.001391452,"about_ca_topic_score_gemma":0.01668037,"domain_scores_codex":[0.9932964,0.001427215,0.001141374,0.0008028299,0.001637819,0.001694398],"domain_scores_gemma":[0.9965624,0.0002015191,0.0005398171,0.0003396506,0.0004023359,0.001954298],"domain_codex":null,"domain_gemma":null,"domain_candidate":null,"domain_consensus":null,"study_design_codex":"observational","study_design_gemma":"observational","study_design_scores_codex":[0.0005207274,0.0001869045,0.9846997,0.00001541013,0.00004082664,0.007355478,0.000331332,0.001519972,0.0002644614,0.00007579236,0.0001060344,0.004883329],"study_design_scores_gemma":[0.003033639,0.2153821,0.7570916,0.00007513203,0.00005615304,0.01647416,0.0007426923,0.001402333,0.0002245214,0.002004184,0.003107526,0.0004059247],"study_design_candidate":"observational","study_design_consensus":"observational","genre_codex":"empirical","genre_gemma":"empirical","genre_scores_codex":[0.9910482,0.002893717,0.00001273911,0.003479707,0.0006030463,0.000985346,0.00000338917,0.00001523074,0.0009585925],"genre_scores_gemma":[0.9952226,0.001009689,0.000664026,0.002846659,0.0001845749,0.00001050167,3.491893e-7,0.0000205045,0.00004106843],"genre_candidate":"empirical","genre_consensus":"empirical","teacher_disagreement_score":0.2276081,"threshold_uncertainty_score":0.9999693,"prediction_status":"machine_predicted_unvalidated"},"machine_scores":{"provisional":true,"baseline":true,"maturity_gate_passed":false,"score_opus":0.0320504291352658,"score_gpt":0.3079442969156574,"score_spread":0.2758938677803916,"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."}}