{"id":"W4389635735","doi":"10.1016/j.ebiom.2023.104894","title":"Effects of gene replacement therapy with resamirigene bilparvovec (AT132) on skeletal muscle pathology in X-linked myotubular myopathy: results from a substudy of the ASPIRO open-label clinical trial","year":2023,"lang":"en","type":"article","venue":"EBioMedicine","topic":"Cellular transport and secretion","field":"Biochemistry, Genetics and Molecular Biology","cited_by":10,"is_retracted":false,"has_abstract":true,"ca_institutions":"Hospital for Sick Children","funders":"National Institute of Neurological Disorders and Stroke; Canadian Institutes of Health Research; Genentech; Grifols; PTC Therapeutics; Audentes Therapeutics; CSL Behring; National Institutes of Health; Argenx; Biogen; Sarepta Therapeutics; FibroGen; Astellas Pharma; Alexion Pharmaceuticals; Pfizer","keywords":"Medicine; Congenital myopathy; Clinical trial; Skeletal muscle; Biopsy; Muscle biopsy; Myopathy; Pathology; Internal medicine","routes":{"ca_aff":true,"ca_fund":true,"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":[0.00162582,0.00062242,0.001578949,0.0003357804,0.0002749321,0.0005495696,0.0004056987,0.0007718289,0.001995583],"category_scores_gemma":[0.0006686469,0.0001715092,0.00129132,0.0003646841,0.0004420204,0.0004315455,0.0001878393,0.001050218,0.000344588],"about_ca_system_candidate":false,"about_ca_system_consensus":false,"about_ca_system_score_codex":0.0005598387,"about_ca_system_score_gemma":0.0004679182,"about_ca_topic_candidate":false,"about_ca_topic_consensus":false,"about_ca_topic_score_codex":0.0006507954,"about_ca_topic_score_gemma":0.002173539,"domain_scores_codex":[0.9995438,0.000228648,0.00003948726,0.00004971201,0.00007202121,0.00006635075],"domain_scores_gemma":[0.9994633,0.0001506554,0.0001545071,0.00004245705,0.0000594269,0.0001296495],"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":[0.6270214,0.02480083,0.01019973,0.004791185,0.005636497,0.0002867223,0.000470161,0.00225208,0.1169332,0.0004233833,0.004177495,0.2030073],"study_design_scores_gemma":[0.2300321,0.7076927,0.03504897,0.0003726849,0.00372535,0.0005196939,0.0001770765,0.001086418,0.01477209,0.0002992856,0.006213694,0.00005996717],"study_design_candidate":"observational","study_design_consensus":null,"genre_codex":"empirical","genre_gemma":"empirical","genre_scores_codex":[0.9861569,0.01041037,0.0002728697,0.0002056943,0.00007908606,0.0006707407,0.0008150238,0.000040608,0.001348609],"genre_scores_gemma":[0.990819,0.004607568,0.0006976715,0.0002977216,0.0001127263,0.0009627568,0.001258301,0.0000140623,0.001230136],"genre_candidate":"empirical","genre_consensus":"empirical","teacher_disagreement_score":0.001995583,"threshold_uncertainty_score":0.008598268,"prediction_status":"machine_predicted_unvalidated"},"machine_scores":{"provisional":true,"baseline":true,"maturity_gate_passed":false,"score_opus":0.04179325689814033,"score_gpt":0.3215600850312476,"score_spread":0.2797668281331073,"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."}}