{"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":"codex-gemma-dda1882f352a","candidate_categories":[],"consensus_categories":[],"category_scores_codex":[0.001038333,0.0001644785,0.0003789273,0.0001664222,0.00006056031,0.00001077538,0.0002941564,0.00005686884,0.00004522058],"category_scores_gemma":[0.000214447,0.0001619428,0.0005289251,0.00009779266,0.00004042598,0.000009335396,0.00008225172,0.0003163134,3.182599e-7],"about_ca_system_candidate":false,"about_ca_system_consensus":false,"about_ca_system_score_codex":0.0001319755,"about_ca_system_score_gemma":0.0002847948,"about_ca_topic_candidate":false,"about_ca_topic_consensus":false,"about_ca_topic_score_codex":0.00001098361,"about_ca_topic_score_gemma":0.0000284045,"domain_scores_codex":[0.9975471,0.0004986289,0.0009724498,0.0003549517,0.0004068165,0.0002200654],"domain_scores_gemma":[0.9991001,0.00003580628,0.0003648117,0.0003029,0.00007849505,0.0001179206],"domain_codex":null,"domain_gemma":null,"domain_candidate":null,"domain_consensus":null,"study_design_codex":"observational","study_design_gemma":"observational","study_design_scores_codex":[0.2406722,0.01652073,0.4366017,0.00009164007,0.0004703926,0.002182169,0.0006427331,0.002809914,0.2961793,0.00001453083,0.001394575,0.002420114],"study_design_scores_gemma":[0.1732806,0.03183212,0.6221163,0.00003155941,0.000559376,0.00009052069,0.001131457,0.0006729436,0.000736325,0.00008141495,0.168727,0.0007403396],"study_design_candidate":"observational","study_design_consensus":"observational","genre_codex":"empirical","genre_gemma":"empirical","genre_scores_codex":[0.9906386,0.006349965,0.000254937,0.00007738165,0.001717348,0.0009345761,0.00001901711,0.00000396111,0.000004205725],"genre_scores_gemma":[0.998553,0.0002032458,0.00006746817,0.0001789892,0.0004944827,0.00007616156,0.0003362841,0.00002692888,0.00006345844],"genre_candidate":"empirical","genre_consensus":"empirical","teacher_disagreement_score":0.2954429,"threshold_uncertainty_score":0.6603836,"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."}}