{"id":"W4281748979","doi":"10.3233/jnd-210781","title":"INCEPTUS Natural History, Run-in Study for Gene Replacement Clinical Trial in X-Linked Myotubular Myopathy","year":2022,"lang":"en","type":"article","venue":"Journal of Neuromuscular Diseases","topic":"Cellular transport and secretion","field":"Biochemistry, Genetics and Molecular Biology","cited_by":25,"is_retracted":false,"has_abstract":true,"ca_institutions":"Hospital for Sick Children","funders":"Eunice Kennedy Shriver National Institute of Child Health and Human Development; Astellas Pharma; Myotubular Trust; Joshua Frase Foundation","keywords":"Medicine; Gastrostomy; Respiratory failure; Prospective cohort study; Adverse effect; Pediatrics; Myopathy; Internal medicine; Surgery","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":[0.0008650759,0.0003510729,0.0006189611,0.0001729498,0.0002826494,0.0006084086,0.0002707563,0.0006039272,0.001981744],"category_scores_gemma":[0.0008174333,0.000112688,0.0004054839,0.0001800565,0.0002676073,0.0002883042,0.0001828041,0.000698275,0.0002016022],"about_ca_system_candidate":false,"about_ca_system_consensus":false,"about_ca_system_score_codex":0.0003240105,"about_ca_system_score_gemma":0.0005478034,"about_ca_topic_candidate":false,"about_ca_topic_consensus":false,"about_ca_topic_score_codex":0.0002879301,"about_ca_topic_score_gemma":0.0008297849,"domain_scores_codex":[0.9997316,0.0001267606,0.00001987262,0.00004457018,0.00002607249,0.00005105851],"domain_scores_gemma":[0.9995036,0.00009816932,0.0001446377,0.00002598407,0.00003178662,0.0001957084],"domain_codex":null,"domain_gemma":null,"domain_candidate":null,"domain_consensus":null,"study_design_codex":"randomized_trial","study_design_gemma":"observational","study_design_scores_codex":[0.7883915,0.01141583,0.103355,0.001033423,0.001307799,0.0005750909,0.0002997923,0.0004276749,0.01067996,0.0005932846,0.002199183,0.07972144],"study_design_scores_gemma":[0.4404682,0.308133,0.2386401,0.0003815842,0.001631797,0.001113519,0.0002434663,0.001704935,0.001905658,0.0006193392,0.005122855,0.00003555584],"study_design_candidate":"observational","study_design_consensus":null,"genre_codex":"empirical","genre_gemma":"empirical","genre_scores_codex":[0.9961473,0.002220518,0.00007739961,0.0002080632,0.00004747086,0.0002081656,0.0001871385,0.00001149219,0.0008924101],"genre_scores_gemma":[0.9966244,0.0006539958,0.0003590378,0.0003818003,0.0001040811,0.0006143603,0.000396233,0.000002994673,0.0008631201],"genre_candidate":"empirical","genre_consensus":"empirical","teacher_disagreement_score":0.001981744,"threshold_uncertainty_score":0.006629586,"prediction_status":"machine_predicted_unvalidated"},"machine_scores":{"provisional":true,"baseline":true,"maturity_gate_passed":false,"score_opus":0.0316516693999498,"score_gpt":0.2989125765968262,"score_spread":0.2672609071968765,"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."}}