{"id":"W3087021087","doi":"10.1002/jimd.12315","title":"Developments in evidence creation for treatments of inborn errors of metabolism","year":2020,"lang":"en","type":"review","venue":"Journal of Inherited Metabolic Disease","topic":"Metabolism and Genetic Disorders","field":"Biochemistry, Genetics and Molecular Biology","cited_by":27,"is_retracted":false,"has_abstract":true,"ca_institutions":"University of Ottawa; BC Children's Hospital; University of British Columbia","funders":"Stichting Metakids; Ultragenyx Pharmaceutical; Canadian Institutes of Health Research; Amicus Therapeutics; BC Children's Hospital; Vitaflo","keywords":"Observational study; Clinical trial; Medicine; Sample size determination; Enzyme replacement therapy; Intensive care medicine; Randomized controlled trial; Transplantation; Clinical study design; Surgery; Internal medicine; Disease","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":[0.005615104,0.001029637,0.002553483,0.004598305,0.0003882404,0.002190062,0.001977765,0.003045059,0.005925283],"category_scores_gemma":[0.00983032,0.0004541284,0.001812136,0.003472779,0.001248324,0.003005007,0.001639773,0.005919534,0.001761388],"about_ca_system_candidate":false,"about_ca_system_consensus":false,"about_ca_system_score_codex":0.001991155,"about_ca_system_score_gemma":0.004027875,"about_ca_topic_candidate":false,"about_ca_topic_consensus":false,"about_ca_topic_score_codex":0.001307329,"about_ca_topic_score_gemma":0.002101221,"domain_scores_codex":[0.9985812,0.0005135093,0.0002374156,0.0001478785,0.0004386275,0.00008146038],"domain_scores_gemma":[0.988407,0.009401845,0.0005776919,0.0001889745,0.00110855,0.0003160405],"domain_codex":null,"domain_gemma":null,"domain_candidate":null,"domain_consensus":null,"study_design_codex":"design_other","study_design_gemma":"not_applicable","study_design_scores_codex":[0.0001008117,0.00004980974,0.00009762156,0.03983956,0.0002915453,0.000190434,0.00007581849,0.0003710128,0.0004480535,0.01770945,0.03058547,0.9102404],"study_design_scores_gemma":[0.00005501788,0.00008399016,0.0005440066,0.04062722,0.0003374557,0.0006908965,0.0000833534,0.0001119717,0.0002108148,0.01582729,0.9413974,0.00003046698],"study_design_candidate":"not_applicable","study_design_consensus":null,"genre_codex":"review","genre_gemma":"review","genre_scores_codex":[0.00003148931,0.9972568,0.0002747601,0.001293261,0.0004453114,0.00001042645,0.00001815668,0.000009171956,0.0006605326],"genre_scores_gemma":[0.0004289848,0.9972869,0.0007790983,0.0008727984,0.0003527709,0.00001964089,0.0000273707,0.000003660277,0.0002287742],"genre_candidate":"review","genre_consensus":"review","teacher_disagreement_score":0.005925283,"threshold_uncertainty_score":0.02969587,"prediction_status":"machine_predicted_unvalidated"},"machine_scores":{"provisional":true,"baseline":true,"maturity_gate_passed":false,"score_opus":0.05335024161605655,"score_gpt":0.3668258170977105,"score_spread":0.3134755754816539,"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."}}