{"id":"W4417123253","doi":"10.1038/s41591-025-04119-2","title":"Intrathecal onasemnogene abeparvovec for treatment-experienced patients with spinal muscular atrophy: a phase 3b, open-label trial","year":2025,"lang":"en","type":"article","venue":"Nature Medicine","topic":"Neurogenetic and Muscular Disorders Research","field":"Medicine","cited_by":10,"is_retracted":false,"has_abstract":true,"ca_institutions":"McGill University; McGill University Health Centre","funders":"Novartis Pharma; Novartis","keywords":"Discontinuation; Adverse effect; Vomiting; Spinal muscular atrophy; Nausea; Weakness; Respiratory system; Diarrhea","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":[0.0009346063,0.0005856468,0.001026785,0.0001550647,0.0002531383,0.0006798083,0.0004803352,0.0009248088,0.003435388],"category_scores_gemma":[0.0005129324,0.0001937703,0.0008759187,0.0001829546,0.0003990002,0.0005115818,0.0001749619,0.001936847,0.0004926259],"about_ca_system_candidate":false,"about_ca_system_consensus":false,"about_ca_system_score_codex":0.0006102543,"about_ca_system_score_gemma":0.0008173298,"about_ca_topic_candidate":false,"about_ca_topic_consensus":false,"about_ca_topic_score_codex":0.0007603401,"about_ca_topic_score_gemma":0.003654142,"domain_scores_codex":[0.9997929,0.0001059411,0.0000100847,0.00002802933,0.00001954165,0.00004352058],"domain_scores_gemma":[0.9997198,0.00006273315,0.00006185641,0.00001278404,0.00002626772,0.0001166314],"domain_codex":null,"domain_gemma":null,"domain_candidate":null,"domain_consensus":null,"study_design_codex":"randomized_trial","study_design_gemma":"nonrandomized_trial","study_design_scores_codex":[0.6387034,0.04432947,0.005617058,0.003296642,0.002716545,0.0003666675,0.0005195244,0.001640467,0.04945421,0.0007402212,0.00787139,0.2447444],"study_design_scores_gemma":[0.5746372,0.3994368,0.01109456,0.0002557175,0.0007931206,0.0004101515,0.0001669506,0.001931395,0.003500313,0.0003751293,0.00735592,0.00004267165],"study_design_candidate":"nonrandomized_trial","study_design_consensus":null,"genre_codex":"empirical","genre_gemma":"empirical","genre_scores_codex":[0.9874306,0.007129899,0.0004277123,0.0005213086,0.0001836539,0.001140632,0.0005724502,0.00007065007,0.002523091],"genre_scores_gemma":[0.9896992,0.003902998,0.0009983373,0.001096896,0.0001570267,0.001344787,0.0007503299,0.00001460803,0.002035772],"genre_candidate":"empirical","genre_consensus":"empirical","teacher_disagreement_score":0.003435388,"threshold_uncertainty_score":0.01149249,"prediction_status":"machine_predicted_unvalidated"},"machine_scores":{"provisional":true,"baseline":true,"maturity_gate_passed":false,"score_opus":0.03435474372342092,"score_gpt":0.4393763893802446,"score_spread":0.4050216456568237,"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."}}