{"id":"W4309329712","doi":"10.1007/978-1-0716-2772-3_2","title":"Viltolarsen: From Preclinical Studies to FDA Approval","year":2022,"lang":"en","type":"article","venue":"Methods in molecular biology","topic":"Muscle Physiology and Disorders","field":"Biochemistry, Genetics and Molecular Biology","cited_by":32,"is_retracted":false,"has_abstract":false,"ca_institutions":"Muscular Dystrophy Canada; University of Alberta","funders":"","keywords":"Medicine; Clinical trial; Exon skipping; Duchenne muscular dystrophy; Food and drug administration; Dystrophin; Clinical research; Population; Muscular dystrophy; Exon; Bioinformatics; Oncology; Pharmacology; Internal medicine; Gene; Genetics; Biology","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":[0.003314452,0.00101272,0.001199009,0.001169447,0.0005379974,0.00327626,0.00111514,0.003645786,0.02095412],"category_scores_gemma":[0.002169693,0.0004579168,0.001053575,0.0005849793,0.001472384,0.001466981,0.0009966418,0.005488881,0.009913941],"about_ca_system_candidate":false,"about_ca_system_consensus":false,"about_ca_system_score_codex":0.001047291,"about_ca_system_score_gemma":0.002741158,"about_ca_topic_candidate":false,"about_ca_topic_consensus":false,"about_ca_topic_score_codex":0.0003878682,"about_ca_topic_score_gemma":0.001212195,"domain_scores_codex":[0.998951,0.0002578881,0.00006016948,0.0001463312,0.0003803833,0.0002043028],"domain_scores_gemma":[0.9986192,0.0002697151,0.0002156427,0.0001298745,0.000254285,0.0005113136],"domain_codex":null,"domain_gemma":null,"domain_candidate":null,"domain_consensus":null,"study_design_codex":"design_other","study_design_gemma":"not_applicable","study_design_scores_codex":[0.004982037,0.001265616,0.00080212,0.002316485,0.0001910504,0.0008465713,0.0001896239,0.0004884378,0.02490013,0.0116245,0.1476124,0.804781],"study_design_scores_gemma":[0.009514475,0.01492052,0.003847616,0.002572341,0.0003074443,0.002578403,0.0001346374,0.0007055815,0.02219094,0.005905796,0.9372007,0.0001214961],"study_design_candidate":"not_applicable","study_design_consensus":null,"genre_codex":"review","genre_gemma":"empirical","genre_scores_codex":[0.1071262,0.3860506,0.01761722,0.09991881,0.0205811,0.002923767,0.01162019,0.007302467,0.3468596],"genre_scores_gemma":[0.4222923,0.2344757,0.02527717,0.07658271,0.005287506,0.001833761,0.01669185,0.001484478,0.2160745],"genre_candidate":"empirical","genre_consensus":null,"teacher_disagreement_score":0.02095412,"threshold_uncertainty_score":0.07009858,"prediction_status":"machine_predicted_unvalidated"},"machine_scores":{"provisional":true,"baseline":true,"maturity_gate_passed":false,"score_opus":0.05641774302297121,"score_gpt":0.4699593189552221,"score_spread":0.4135415759322509,"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."}}