{"id":"W4391708105","doi":"10.1212/wnl.0000000000208112","title":"Efficacy and Safety of Vamorolone Over 48 Weeks in Boys With Duchenne Muscular Dystrophy","year":2024,"lang":"en","type":"article","venue":"Neurology","topic":"Muscle Physiology and Disorders","field":"Biochemistry, Genetics and Molecular Biology","cited_by":70,"is_retracted":false,"has_abstract":true,"ca_institutions":"Veterans Affairs Canada","funders":"National Center for Advancing Translational Sciences; Duchenne Research Fund; National Institute of Arthritis and Musculoskeletal and Skin Diseases; National Institutes of Health; National Institute for Health and Care Research; Parent Project Muscular Dystrophy; National Institute of Neurological Disorders and Stroke; Foundation to Eradicate Duchenne; Children's Trust; Eunice Kennedy Shriver National Institute of Child Health and Human Development; European Commission; Muscular Dystrophy Association","keywords":"Duchenne muscular dystrophy; Medicine; Prednisone; Crossover study; Placebo; Adverse effect; Supine position; Muscular dystrophy; Ambulatory; Randomized controlled trial; Internal medicine; Anesthesia; Physical therapy","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":[0.00007526696,0.0001210788,0.0001696199,0.00005953757,0.00002101163,0.000004136757,0.0000831064,0.0001811541,0.00003090556],"category_scores_gemma":[0.00002415982,0.0001021476,0.00003562937,0.00008894506,0.0002022583,0.000003162846,0.00007008752,0.0001658335,0.000003931659],"about_ca_system_candidate":false,"about_ca_system_consensus":false,"about_ca_system_score_codex":0.000001662111,"about_ca_system_score_gemma":0.00003362902,"about_ca_topic_candidate":false,"about_ca_topic_consensus":false,"about_ca_topic_score_codex":0.00005917138,"about_ca_topic_score_gemma":0.0000782914,"domain_scores_codex":[0.9991635,0.00008575548,0.0001462336,0.0003675415,0.00004438667,0.000192578],"domain_scores_gemma":[0.9996796,0.00002778498,0.00002819485,0.0002119145,0.00001426371,0.00003824246],"domain_codex":null,"domain_gemma":null,"domain_candidate":null,"domain_consensus":null,"study_design_codex":"bench_or_experimental","study_design_gemma":"observational","study_design_scores_codex":[0.01231676,0.0002750676,0.01638528,0.0001712429,0.0002101906,0.0001608647,0.0001374711,0.0008211515,0.9625921,0.001884467,0.0009059392,0.004139525],"study_design_scores_gemma":[0.00743493,0.008983861,0.9073188,0.00003510051,0.0001002808,0.0001607939,0.00001611955,0.0007458526,0.01401937,0.0009713567,0.0596931,0.0005203906],"study_design_candidate":"bench_or_experimental","study_design_consensus":null,"genre_codex":"empirical","genre_gemma":"empirical","genre_scores_codex":[0.9973853,0.001359562,0.0000964564,0.0004950095,0.0001064083,0.0001312265,0.000008357873,0.00001109666,0.0004065363],"genre_scores_gemma":[0.9990626,0.0002540208,0.00004228598,0.0004974777,0.00005733178,0.00001128187,0.00003233314,0.00001631568,0.00002636536],"genre_candidate":"empirical","genre_consensus":"empirical","teacher_disagreement_score":0.9485726,"threshold_uncertainty_score":0.4165457,"prediction_status":"machine_predicted_unvalidated"},"machine_scores":{"provisional":true,"baseline":true,"maturity_gate_passed":false,"score_opus":0.003537335085469933,"score_gpt":0.2139212626585576,"score_spread":0.2103839275730876,"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."}}