{"id":"W3170463959","doi":"10.1017/cjn.2019.90","title":"B.01 AVXS-101 gene-replacement therapy (GRT) for spinal muscular atrophy type 1 (SMA1): pivotal phase 3 study (STR1VE) 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":2,"is_retracted":false,"has_abstract":true,"ca_institutions":"","funders":"","keywords":"SMN1; Medicine; Internal medicine; Toddler; Spinal muscular atrophy; Pediatrics; Disease; Psychology","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":["sts"],"category_scores_codex":[0.004329274,0.000437697,0.0007699467,0.0009656301,0.001744999,0.0005274767,0.001655933,0.000172008,0.0005647377],"category_scores_gemma":[0.0009599053,0.0002873261,0.0004843844,0.00132431,0.002766945,0.0004829005,0.00008578895,0.001160069,0.00001547181],"about_ca_system_candidate":false,"about_ca_system_consensus":false,"about_ca_system_score_codex":0.0002030589,"about_ca_system_score_gemma":0.004647943,"about_ca_topic_candidate":false,"about_ca_topic_consensus":false,"about_ca_topic_score_codex":0.0005326245,"about_ca_topic_score_gemma":0.004620996,"domain_scores_codex":[0.9942308,0.0008269422,0.0009943303,0.0008497635,0.001389728,0.001708422],"domain_scores_gemma":[0.9958068,0.000226989,0.0005690736,0.0003216536,0.0007910058,0.002284486],"domain_codex":null,"domain_gemma":null,"domain_candidate":null,"domain_consensus":null,"study_design_codex":"observational","study_design_gemma":"nonrandomized_trial","study_design_scores_codex":[0.004486802,0.0007507802,0.9646878,0.00003348548,0.0001673665,0.007352292,0.0004356749,0.0009626109,0.001533712,0.000247349,0.001078738,0.01826342],"study_design_scores_gemma":[0.00917764,0.794519,0.1402418,0.00005988777,0.000182205,0.02268274,0.001064406,0.002149399,0.0009104001,0.002175405,0.02613818,0.0006989565],"study_design_candidate":"observational","study_design_consensus":null,"genre_codex":"empirical","genre_gemma":"empirical","genre_scores_codex":[0.9919912,0.002393428,0.00007300806,0.002661826,0.001088413,0.001286182,0.0000124812,0.00001839257,0.0004750459],"genre_scores_gemma":[0.9939304,0.0007762221,0.001405502,0.00343976,0.0003401107,0.00001195985,0.000001200986,0.00002619071,0.00006865281],"genre_candidate":"empirical","genre_consensus":"empirical","teacher_disagreement_score":0.824446,"threshold_uncertainty_score":0.9999579,"prediction_status":"machine_predicted_unvalidated"},"machine_scores":{"provisional":true,"baseline":true,"maturity_gate_passed":false,"score_opus":0.05149592150411585,"score_gpt":0.3476042676627457,"score_spread":0.2961083461586298,"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."}}