{"id":"W4398781241","doi":"10.1017/cjn.2024.95","title":"D.4 Safety and efficacy of delandistrogene moxeparvovec versus placebo in Duchenne muscular dystrophy (EMBARK): Pivotal Phase 3 primary results","year":2024,"lang":"en","type":"article","venue":"Canadian Journal of Neurological Sciences / Journal Canadien des Sciences Neurologiques","topic":"Muscle Physiology and Disorders","field":"Biochemistry, Genetics and Molecular Biology","cited_by":0,"is_retracted":false,"has_abstract":true,"ca_institutions":"","funders":"","keywords":"Duchenne muscular dystrophy; Clinical endpoint; Medicine; Placebo; Ambulatory; Clinical trial; Physical therapy; Internal medicine; Dystrophin; Pediatrics; Pathology","routes":{"ca_aff":false,"ca_fund":false,"ca_venue":true,"about_ca":false,"invisible_to_affiliation_only":true},"retraction":null,"screen":null,"direct_labels":[],"prediction":{"model_version":"metacan-v3-hybrid-931329e0061c","candidate_categories":[],"consensus_categories":[],"category_scores_codex":[0.001579213,0.0011006,0.001397874,0.0003082067,0.0003322604,0.001055261,0.0007116778,0.001594532,0.0104839],"category_scores_gemma":[0.001204839,0.0003814586,0.001230047,0.0002639611,0.0005270655,0.0005866141,0.0001811764,0.002009796,0.001211125],"about_ca_system_candidate":false,"about_ca_system_consensus":false,"about_ca_system_score_codex":0.0006484073,"about_ca_system_score_gemma":0.0009083482,"about_ca_topic_candidate":false,"about_ca_topic_consensus":false,"about_ca_topic_score_codex":0.0007092587,"about_ca_topic_score_gemma":0.002192997,"domain_scores_codex":[0.9995895,0.0002182148,0.00002720857,0.00005210389,0.00003831673,0.00007465218],"domain_scores_gemma":[0.9994268,0.0001584019,0.0001759799,0.00003096363,0.00005318271,0.0001547682],"domain_codex":null,"domain_gemma":null,"domain_candidate":null,"domain_consensus":null,"study_design_codex":"randomized_trial","study_design_gemma":"randomized_trial","study_design_scores_codex":[0.9147819,0.01085409,0.000857891,0.001561396,0.0008290624,0.0001101739,0.00008351207,0.0005766302,0.02110606,0.0002822427,0.005827198,0.04312976],"study_design_scores_gemma":[0.7475957,0.2385731,0.004656441,0.0001618503,0.0006133835,0.0001406358,0.00004475273,0.0006022299,0.004032218,0.0002055591,0.003346912,0.00002720424],"study_design_candidate":"randomized_trial","study_design_consensus":"randomized_trial","genre_codex":"empirical","genre_gemma":"empirical","genre_scores_codex":[0.9621928,0.01450876,0.001140224,0.002014428,0.0006161378,0.004964546,0.005500293,0.0003274862,0.008735231],"genre_scores_gemma":[0.9745944,0.004838391,0.001853222,0.001838135,0.0004690509,0.00463993,0.002636637,0.00004695611,0.009083382],"genre_candidate":"empirical","genre_consensus":"empirical","teacher_disagreement_score":0.0104839,"threshold_uncertainty_score":0.03507209,"prediction_status":"machine_predicted_unvalidated"},"machine_scores":{"provisional":true,"baseline":true,"maturity_gate_passed":false,"score_opus":0.01561187381896112,"score_gpt":0.2668989862403439,"score_spread":0.2512871124213827,"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."}}