{"id":"W2264063527","doi":"10.1016/j.ymgmr.2017.10.008","title":"Long-term outcomes with agalsidase alfa enzyme replacement therapy: Analysis using deconstructed composite events","year":2017,"lang":"en","type":"article","venue":"Molecular Genetics and Metabolism Reports","topic":"Lysosomal Storage Disorders Research","field":"Medicine","cited_by":15,"is_retracted":false,"has_abstract":true,"ca_institutions":"Dalhousie University","funders":"Amicus Therapeutics; Alexion Pharmaceuticals; Sanofi; GlaxoSmithKline","keywords":"Enzyme replacement therapy; Medicine; Fabry disease; Renal replacement therapy; Internal medicine; Young adult; Pediatrics; Cardiology; Disease","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":[0.003756854,0.0004445428,0.0008359991,0.00150486,0.0002602363,0.0008619185,0.0004132371,0.0003281867,0.0007490074],"category_scores_gemma":[0.006878714,0.000157716,0.001827683,0.001408972,0.0002914311,0.0007215068,0.0009946912,0.0007161286,0.0001337942],"about_ca_system_candidate":false,"about_ca_system_consensus":false,"about_ca_system_score_codex":0.0002924454,"about_ca_system_score_gemma":0.0004237811,"about_ca_topic_candidate":false,"about_ca_topic_consensus":false,"about_ca_topic_score_codex":0.001500268,"about_ca_topic_score_gemma":0.001881452,"domain_scores_codex":[0.9981382,0.000706709,0.0002905314,0.0002490028,0.0004013172,0.0002142099],"domain_scores_gemma":[0.9920211,0.002769831,0.003384613,0.0006441588,0.0005908972,0.0005892807],"domain_codex":null,"domain_gemma":null,"domain_candidate":null,"domain_consensus":null,"study_design_codex":"observational","study_design_gemma":"observational","study_design_scores_codex":[0.001135143,0.00002965764,0.9955904,0.00002345857,0.0006417405,0.00004923845,0.00003350679,0.0003515875,0.0001480381,0.00003830191,0.00009525698,0.001863662],"study_design_scores_gemma":[0.00002641444,0.0005219643,0.9978638,0.000009019207,0.0002302524,0.000170905,0.00007634981,0.0007356547,0.0001026749,0.00005094859,0.0002026662,0.000009292472],"study_design_candidate":"observational","study_design_consensus":"observational","genre_codex":"empirical","genre_gemma":"empirical","genre_scores_codex":[0.9972489,0.0003738832,0.0004263929,0.00001962,0.000009014587,0.0000182506,0.001559958,0.000006871828,0.0003369813],"genre_scores_gemma":[0.9965019,0.0001073535,0.0002420531,0.0000119715,0.00001894534,0.00002981064,0.002993811,0.000005396781,0.00008870232],"genre_candidate":"empirical","genre_consensus":"empirical","teacher_disagreement_score":0.003756854,"threshold_uncertainty_score":0.01986837,"prediction_status":"machine_predicted_unvalidated"},"machine_scores":{"provisional":true,"baseline":true,"maturity_gate_passed":false,"score_opus":0.02680971996918637,"score_gpt":0.3285412045545899,"score_spread":0.3017314845854036,"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."}}