{"id":"W4283031673","doi":"10.1038/s41591-022-01867-3","title":"Onasemnogene abeparvovec for presymptomatic infants with three copies of SMN2 at risk for spinal muscular atrophy: the Phase III SPR1NT trial","year":2022,"lang":"en","type":"article","venue":"Nature Medicine","topic":"Neurogenetic and Muscular Disorders Research","field":"Medicine","cited_by":276,"is_retracted":false,"has_abstract":true,"ca_institutions":"McGill University; Montreal Children's Hospital","funders":"Novartis Institutes for BioMedical Research; Genentech; New South Wales Government; University of Sydney; Sarepta Therapeutics; University of New South Wales; Biogen; Children's Medical Research","keywords":"Spinal muscular atrophy; Medicine; Atrophy; Pediatrics; Internal medicine; 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":[0.0005137887,0.000383328,0.0005361185,0.00009648458,0.0001354781,0.0003783332,0.0002907465,0.0004995717,0.002007944],"category_scores_gemma":[0.0003631127,0.0000973654,0.0004709121,0.0001000526,0.0001677252,0.0002262369,0.0001357669,0.001063806,0.0002361381],"about_ca_system_candidate":false,"about_ca_system_consensus":false,"about_ca_system_score_codex":0.0003524353,"about_ca_system_score_gemma":0.0004624682,"about_ca_topic_candidate":false,"about_ca_topic_consensus":false,"about_ca_topic_score_codex":0.0007880161,"about_ca_topic_score_gemma":0.004064916,"domain_scores_codex":[0.9998707,0.00006495413,0.000005058288,0.00001573111,0.00001900451,0.00002473876],"domain_scores_gemma":[0.9998536,0.00004620434,0.00002845091,0.000007043987,0.00001276571,0.00005190536],"domain_codex":null,"domain_gemma":null,"domain_candidate":null,"domain_consensus":null,"study_design_codex":"design_other","study_design_gemma":"randomized_trial","study_design_scores_codex":[0.3959935,0.01299838,0.01239568,0.002475285,0.002257767,0.0009702473,0.0003834408,0.002222855,0.0860412,0.001809032,0.01823396,0.4642187],"study_design_scores_gemma":[0.5370067,0.3142225,0.07303224,0.000841085,0.001679032,0.002409908,0.000365177,0.004414026,0.02370224,0.001436417,0.04081798,0.00007269241],"study_design_candidate":"randomized_trial","study_design_consensus":null,"genre_codex":"empirical","genre_gemma":"empirical","genre_scores_codex":[0.982251,0.01174864,0.0003688149,0.0007051423,0.0001754687,0.0003164781,0.0008467608,0.00007531825,0.003512386],"genre_scores_gemma":[0.9853536,0.006907534,0.001163794,0.0008820046,0.00008875224,0.0004473784,0.00134356,0.00002311122,0.003790211],"genre_candidate":"empirical","genre_consensus":"empirical","teacher_disagreement_score":0.002007944,"threshold_uncertainty_score":0.006717205,"prediction_status":"machine_predicted_unvalidated"},"machine_scores":{"provisional":true,"baseline":true,"maturity_gate_passed":false,"score_opus":0.02039266236607381,"score_gpt":0.3519006474677333,"score_spread":0.3315079851016595,"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."}}