{"id":"W3047556542","doi":"10.1358/dot.2020.56.8.3159186","title":"Golodirsen for Duchenne muscular dystrophy","year":2020,"lang":"en","type":"article","venue":"Drugs of today","topic":"Muscle Physiology and Disorders","field":"Biochemistry, Genetics and Molecular Biology","cited_by":70,"is_retracted":false,"has_abstract":true,"ca_institutions":"Muscular Dystrophy Canada; University of Alberta","funders":"","keywords":"Exon skipping; Duchenne muscular dystrophy; Exon; Medicine; Dystrophin; Morpholino; Muscular dystrophy; Gene; Bioinformatics; Genetics; Antisense therapy; Internal medicine; Gene knockdown; Biology; Oligonucleotide; Alternative splicing","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":"codex-gemma-dda1882f352a","candidate_categories":[],"consensus_categories":[],"category_scores_codex":[0.00005269606,0.00008847899,0.0001301195,0.00001067867,0.00003172455,0.00000291413,0.0001486539,0.00009416066,0.00002688166],"category_scores_gemma":[0.00009886928,0.00008377368,0.0001353637,0.00003660406,0.00006575133,0.000001891895,0.00005515642,0.00003490421,0.000008031533],"about_ca_system_candidate":false,"about_ca_system_consensus":false,"about_ca_system_score_codex":0.000001609704,"about_ca_system_score_gemma":0.0000255541,"about_ca_topic_candidate":false,"about_ca_topic_consensus":false,"about_ca_topic_score_codex":0.000007620752,"about_ca_topic_score_gemma":0.000001044398,"domain_scores_codex":[0.9994496,0.00002039234,0.0001241169,0.0002202461,0.00004497959,0.0001407046],"domain_scores_gemma":[0.999647,0.00001095656,0.00005395381,0.0001725862,0.00005244076,0.00006308496],"domain_codex":null,"domain_gemma":null,"domain_candidate":null,"domain_consensus":null,"study_design_codex":"bench_or_experimental","study_design_gemma":"bench_or_experimental","study_design_scores_codex":[0.0003932115,0.00004875636,0.0001829293,0.00007461049,0.0000810476,4.087039e-7,0.0001585335,0.0001445136,0.9841633,0.0007432917,0.01204001,0.001969328],"study_design_scores_gemma":[0.002536278,0.00130169,0.001842251,0.00001069151,0.00006264455,0.000001002427,0.0004186043,0.000243114,0.6516961,0.001099039,0.3404003,0.0003883107],"study_design_candidate":"bench_or_experimental","study_design_consensus":"bench_or_experimental","genre_codex":"empirical","genre_gemma":"empirical","genre_scores_codex":[0.9945045,0.000435761,0.001508198,0.001646821,0.00008299381,0.0001958629,0.00004821484,0.00001134768,0.001566375],"genre_scores_gemma":[0.9976798,0.00003502857,0.0009200905,0.0009222474,0.0001781424,0.00002155494,0.000132948,0.00001244543,0.00009768926],"genre_candidate":"empirical","genre_consensus":"empirical","teacher_disagreement_score":0.3324673,"threshold_uncertainty_score":0.3416192,"prediction_status":"machine_predicted_unvalidated"},"machine_scores":{"provisional":true,"baseline":true,"maturity_gate_passed":false,"score_opus":0.0094307765891266,"score_gpt":0.2321511105201688,"score_spread":0.2227203339310422,"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."}}