{"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":"metacan-v3-hybrid-931329e0061c","candidate_categories":[],"consensus_categories":[],"category_scores_codex":[0.001163066,0.0005373048,0.001653734,0.0003662437,0.0003892751,0.000339566,0.0005672851,0.001163438,0.002106171],"category_scores_gemma":[0.001374735,0.0003037225,0.0007923447,0.0002254982,0.0003904096,0.0003862885,0.0002562817,0.001009102,0.0003041687],"about_ca_system_candidate":false,"about_ca_system_consensus":false,"about_ca_system_score_codex":0.0004702321,"about_ca_system_score_gemma":0.0004397553,"about_ca_topic_candidate":false,"about_ca_topic_consensus":false,"about_ca_topic_score_codex":0.0005703146,"about_ca_topic_score_gemma":0.001176539,"domain_scores_codex":[0.9992492,0.0003715823,0.00006236679,0.0001221614,0.0001082005,0.00008649206],"domain_scores_gemma":[0.9989312,0.0002377012,0.0003801183,0.00007138956,0.00008793872,0.0002916706],"domain_codex":null,"domain_gemma":null,"domain_candidate":null,"domain_consensus":null,"study_design_codex":"randomized_trial","study_design_gemma":"nonrandomized_trial","study_design_scores_codex":[0.6035341,0.04587674,0.03468531,0.002316176,0.001605635,0.0004931553,0.0007185852,0.00129972,0.0636685,0.00008478529,0.0008709118,0.2448463],"study_design_scores_gemma":[0.08458123,0.7943701,0.1108745,0.000182288,0.0005097274,0.0004060215,0.0001939435,0.0004268164,0.006742337,0.00005161979,0.001628297,0.00003326283],"study_design_candidate":"nonrandomized_trial","study_design_consensus":null,"genre_codex":"empirical","genre_gemma":"empirical","genre_scores_codex":[0.9965706,0.002788162,0.00007812635,0.00007355527,0.00002720611,0.0001407691,0.00009611978,0.00001972482,0.000205715],"genre_scores_gemma":[0.9976892,0.0009004134,0.0003447846,0.00007079155,0.00006164489,0.0002607021,0.0001383028,0.000004059989,0.0005300327],"genre_candidate":"empirical","genre_consensus":"empirical","teacher_disagreement_score":0.002106171,"threshold_uncertainty_score":0.007045805,"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."}}