{"id":"W4286008638","doi":"10.1093/brain/awac252","title":"Effect of nusinersen on motor, respiratory and bulbar function in early-onset spinal muscular atrophy","year":2022,"lang":"en","type":"article","venue":"Brain","topic":"Neurogenetic and Muscular Disorders Research","field":"Medicine","cited_by":70,"is_retracted":false,"has_abstract":true,"ca_institutions":"Ottawa Hospital; Children's Hospital of Eastern Ontario; University of Ottawa","funders":"Eisai; Albert-Ludwigs-Universität Freiburg; Canada Research Chairs; Muscular Dystrophy Canada; Sarepta Therapeutics; Canadian Institutes of Health Research; GW Pharmaceuticals; Sanofi; PTC Therapeutics; Ultragenyx Pharmaceutical; AveXis; Biogen; Alnylam Pharmaceuticals; F. Hoffmann-La Roche; Alexion Pharmaceuticals; Pfizer","keywords":"Spinal muscular atrophy; Medicine; Pediatrics; Atrophy; Weakness; Progressive muscular atrophy; Neuromuscular disease; Clinical trial; Physical therapy; Physical medicine and rehabilitation; Surgery; Disease; Internal medicine; Amyotrophic lateral sclerosis","routes":{"ca_aff":true,"ca_fund":true,"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":[0.001496827,0.0003204152,0.001061954,0.0004492373,0.0001686286,0.0003495747,0.0002453933,0.0003338789,0.0006303504],"category_scores_gemma":[0.002055183,0.000121801,0.001017642,0.0003357319,0.0002250223,0.0002482521,0.0003227819,0.0003219978,0.00007382026],"about_ca_system_candidate":false,"about_ca_system_consensus":false,"about_ca_system_score_codex":0.0002285189,"about_ca_system_score_gemma":0.0002111794,"about_ca_topic_candidate":false,"about_ca_topic_consensus":false,"about_ca_topic_score_codex":0.0007293827,"about_ca_topic_score_gemma":0.002775621,"domain_scores_codex":[0.9989424,0.000563426,0.0001615675,0.0001153301,0.0001283654,0.0000888603],"domain_scores_gemma":[0.9981158,0.001054804,0.0005143288,0.00009595123,0.00008866797,0.0001304694],"domain_codex":null,"domain_gemma":null,"domain_candidate":null,"domain_consensus":null,"study_design_codex":"observational","study_design_gemma":"randomized_trial","study_design_scores_codex":[0.153376,0.002873126,0.584063,0.004109584,0.009360178,0.001533923,0.0004751025,0.002395494,0.01981672,0.0001898526,0.0008762142,0.2209308],"study_design_scores_gemma":[0.002908519,0.04344537,0.9447023,0.0002229893,0.002106492,0.001070816,0.0001644041,0.0006560876,0.002948632,0.00007756957,0.001670913,0.00002589956],"study_design_candidate":"randomized_trial","study_design_consensus":null,"genre_codex":"empirical","genre_gemma":"empirical","genre_scores_codex":[0.9924957,0.006828618,0.00003925075,0.00002738767,0.000009907284,0.00001517432,0.0002291749,0.000004503931,0.0003501703],"genre_scores_gemma":[0.9982267,0.001223548,0.00008678658,0.0000354123,0.00000751269,0.000009438619,0.0002988068,0.000001312303,0.000110532],"genre_candidate":"empirical","genre_consensus":"empirical","teacher_disagreement_score":0.001496827,"threshold_uncertainty_score":0.007916033,"prediction_status":"machine_predicted_unvalidated"},"machine_scores":{"provisional":true,"baseline":true,"maturity_gate_passed":false,"score_opus":0.01255415004248971,"score_gpt":0.2901318235974736,"score_spread":0.2775776735549839,"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."}}