{"id":"W4283011798","doi":"10.1038/s41591-022-01866-4","title":"Onasemnogene abeparvovec for presymptomatic infants with two copies of SMN2 at risk for spinal muscular atrophy type 1: the Phase III SPR1NT trial","year":2022,"lang":"en","type":"article","venue":"Nature Medicine","topic":"Neurogenetic and Muscular Disorders Research","field":"Medicine","cited_by":295,"is_retracted":false,"has_abstract":true,"ca_institutions":"McGill University Health Centre; Montreal Children's Hospital","funders":"UCB Pharma; Chugai Pharmaceutical; Genentech; Grifols; Novartis Institutes for BioMedical Research; New South Wales Government; University of Sydney; Sarepta Therapeutics; Cytokinetics; PTC Therapeutics; Children's Hospital of Philadelphia; Children's Medical Research; University of New South Wales; Alexion Pharmaceuticals; AveXis; Biogen","keywords":"Spinal muscular atrophy; SMN1; Medicine; SMA*; Pediatrics; Cohort; Clinical trial; Adverse effect; Internal medicine","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":[0.000497556,0.0003776896,0.0005128855,0.0000932283,0.0001395509,0.000356246,0.0002739867,0.0004382619,0.001586228],"category_scores_gemma":[0.000310015,0.00008976216,0.0003778311,0.00007874171,0.0001736526,0.000200144,0.0001381784,0.0009322975,0.0001951241],"about_ca_system_candidate":false,"about_ca_system_consensus":false,"about_ca_system_score_codex":0.0003589121,"about_ca_system_score_gemma":0.0004010088,"about_ca_topic_candidate":false,"about_ca_topic_consensus":false,"about_ca_topic_score_codex":0.0005310521,"about_ca_topic_score_gemma":0.002603273,"domain_scores_codex":[0.9998752,0.00006084697,0.000004951276,0.00001675884,0.0000178498,0.00002427024],"domain_scores_gemma":[0.999862,0.00004185483,0.0000284847,0.000007288512,0.00001143773,0.0000489719],"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":[0.3938123,0.01451085,0.0181824,0.00233666,0.001948506,0.001343262,0.0003746439,0.00245961,0.109937,0.001526998,0.01464104,0.4389267],"study_design_scores_gemma":[0.4242141,0.4011939,0.09370127,0.0007005272,0.001657094,0.003568046,0.0003420984,0.004657938,0.03143879,0.001151803,0.03730396,0.0000705178],"study_design_candidate":"randomized_trial","study_design_consensus":null,"genre_codex":"empirical","genre_gemma":"empirical","genre_scores_codex":[0.9897336,0.006341919,0.0002661073,0.0004028556,0.0001008617,0.000273245,0.000659161,0.00004738786,0.002174781],"genre_scores_gemma":[0.9903853,0.003819619,0.0009577964,0.0006021888,0.00008038792,0.000438833,0.001298125,0.00001585069,0.002401919],"genre_candidate":"empirical","genre_consensus":"empirical","teacher_disagreement_score":0.001586228,"threshold_uncertainty_score":0.005306482,"prediction_status":"machine_predicted_unvalidated"},"machine_scores":{"provisional":true,"baseline":true,"maturity_gate_passed":false,"score_opus":0.02059131339486083,"score_gpt":0.3716390112580729,"score_spread":0.3510476978632121,"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."}}