{"id":"W4387302584","doi":"10.1016/j.nmd.2023.07.027","title":"P46 ENVOL, a Phase 2, open-label trial evaluating the safety and expression of delandistrogene moxeparvovec in Duchenne muscular dystrophy: study design","year":2023,"lang":"en","type":"article","venue":"Neuromuscular Disorders","topic":"Muscle Physiology and Disorders","field":"Biochemistry, Genetics and Molecular Biology","cited_by":0,"is_retracted":false,"has_abstract":false,"ca_institutions":"Roche (Canada)","funders":"","keywords":"Duchenne muscular dystrophy; Open label; Medicine; Muscular dystrophy; Physical medicine and rehabilitation; Internal medicine; Clinical trial; Oncology; 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":[0.001139761,0.0002757343,0.0003605176,0.00009112987,0.0002291711,0.00003452567,0.0005398317,0.000140163,0.00001561526],"category_scores_gemma":[0.0004298002,0.000217648,0.00009835674,0.0003648542,0.000180225,0.00001681875,0.0005113563,0.0001849556,0.000003847236],"about_ca_system_candidate":false,"about_ca_system_consensus":false,"about_ca_system_score_codex":0.000008153657,"about_ca_system_score_gemma":0.00009891249,"about_ca_topic_candidate":false,"about_ca_topic_consensus":false,"about_ca_topic_score_codex":0.0001277218,"about_ca_topic_score_gemma":0.00009430838,"domain_scores_codex":[0.9971839,0.001009324,0.0004754995,0.0006932489,0.0002581718,0.0003798559],"domain_scores_gemma":[0.9989946,0.0001057862,0.0001751791,0.0006001073,0.00004696875,0.00007729808],"domain_codex":null,"domain_gemma":null,"domain_candidate":null,"domain_consensus":null,"study_design_codex":"bench_or_experimental","study_design_gemma":"randomized_trial","study_design_scores_codex":[0.0607167,0.00290917,0.00135555,0.00004860256,0.0001716652,0.00001402491,0.0005552042,0.00586648,0.9188277,0.00003190138,0.0002169461,0.009286052],"study_design_scores_gemma":[0.7722847,0.08019007,0.04833654,0.0001641981,0.0007732554,0.00001924022,0.00787248,0.0304058,0.0502046,0.002964024,0.004202738,0.002582348],"study_design_candidate":"bench_or_experimental","study_design_consensus":null,"genre_codex":"empirical","genre_gemma":"empirical","genre_scores_codex":[0.9955361,0.0005389376,0.001107015,0.0001182477,0.0001154115,0.002491249,0.00002020244,0.00001877815,0.00005398275],"genre_scores_gemma":[0.9987149,0.0005048259,0.0002103045,0.00007328853,0.00004436365,0.0002698922,0.0001132364,0.00003947148,0.00002976492],"genre_candidate":"empirical","genre_consensus":"empirical","teacher_disagreement_score":0.8686231,"threshold_uncertainty_score":0.8875427,"prediction_status":"machine_predicted_unvalidated"},"machine_scores":{"provisional":true,"baseline":true,"maturity_gate_passed":false,"score_opus":0.06171342177315503,"score_gpt":0.3690074144959761,"score_spread":0.307293992722821,"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."}}