{"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":"codex-gemma-dda1882f352a","candidate_categories":[],"consensus_categories":[],"category_scores_codex":[0.001476469,0.0001985758,0.0004926042,0.0001007613,0.00004118493,0.000004974152,0.0004161538,0.0002237302,0.000007853379],"category_scores_gemma":[0.000227957,0.0001250895,0.00008910251,0.0004348519,0.0002777757,0.000003604315,0.00008961884,0.0001750768,0.000002916982],"about_ca_system_candidate":false,"about_ca_system_consensus":false,"about_ca_system_score_codex":0.00001309917,"about_ca_system_score_gemma":0.0001139555,"about_ca_topic_candidate":false,"about_ca_topic_consensus":false,"about_ca_topic_score_codex":0.0002111552,"about_ca_topic_score_gemma":0.0001456979,"domain_scores_codex":[0.9976708,0.0003354588,0.0008448293,0.0005904674,0.0003229443,0.0002354718],"domain_scores_gemma":[0.9985978,0.0001456007,0.0003476389,0.0007725951,0.00007241384,0.00006392112],"domain_codex":null,"domain_gemma":null,"domain_candidate":null,"domain_consensus":null,"study_design_codex":"bench_or_experimental","study_design_gemma":"bench_or_experimental","study_design_scores_codex":[0.04498029,0.001247962,0.002994427,0.00001901366,0.0001057998,0.00006658867,0.0003263836,0.000009740083,0.9478412,0.000006061612,0.0001078209,0.002294688],"study_design_scores_gemma":[0.2168414,0.02165795,0.3530293,0.0001785889,0.00008933603,0.000002422762,0.0001217615,0.00008604478,0.4022051,0.00004631343,0.005491372,0.0002503802],"study_design_candidate":"bench_or_experimental","study_design_consensus":"bench_or_experimental","genre_codex":"empirical","genre_gemma":"empirical","genre_scores_codex":[0.9967156,0.0005086213,0.000160071,0.0003587032,0.0005197878,0.001602435,0.00006819451,0.00001021671,0.00005636552],"genre_scores_gemma":[0.9963439,0.0006774193,0.0001243527,0.0001969786,0.0002827531,0.00005661528,0.002139329,0.00002722931,0.0001514363],"genre_candidate":"empirical","genre_consensus":"empirical","teacher_disagreement_score":0.5456361,"threshold_uncertainty_score":0.5101,"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."}}