{"id":"W3003771398","doi":"10.1016/j.ymgme.2019.11.397","title":"Baseline patient characteristics of followME, a new, patient-centric, prospective, observational Fabry disease registry that evaluates migalastat, ERT, and a natural history cohort","year":2020,"lang":"en","type":"article","venue":"Molecular Genetics and Metabolism","topic":"Lysosomal Storage Disorders Research","field":"Medicine","cited_by":0,"is_retracted":false,"has_abstract":false,"ca_institutions":"Université de Montréal; Hôpital du Sacré-Cœur de Montréal","funders":"","keywords":"Fabry disease; Medicine; Bioequivalence; Pharmacokinetics; Enzyme replacement therapy; Confidence interval; Globotriaosylceramide; Internal medicine; Pharmacodynamics; Urology; Cmax; Alpha-galactosidase; Cohort; Pharmacology; 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":[0.00060462,0.0002892436,0.0004149964,0.0007533984,0.0005305063,0.0006786799,0.0003599418,0.0004202077,0.002062429],"category_scores_gemma":[0.001742962,0.0002435013,0.0003758148,0.001040961,0.0002729386,0.0006955857,0.0004141893,0.0005634409,0.0004124595],"about_ca_system_candidate":false,"about_ca_system_consensus":false,"about_ca_system_score_codex":0.0005068774,"about_ca_system_score_gemma":0.0006325512,"about_ca_topic_candidate":false,"about_ca_topic_consensus":false,"about_ca_topic_score_codex":0.005064064,"about_ca_topic_score_gemma":0.00771201,"domain_scores_codex":[0.9995129,0.00006903155,0.00006037173,0.0001869281,0.00007994195,0.00009073724],"domain_scores_gemma":[0.9988766,0.0001418765,0.0005025779,0.0001744767,0.0001097432,0.0001946125],"domain_codex":null,"domain_gemma":null,"domain_candidate":null,"domain_consensus":null,"study_design_codex":"observational","study_design_gemma":"observational","study_design_scores_codex":[0.0008796211,0.0001188833,0.9943939,0.00001078506,0.0001019775,0.0001501848,0.00008253867,0.0001657801,0.0006580234,0.0001405149,0.001178146,0.002119533],"study_design_scores_gemma":[0.00009053727,0.000321939,0.9965317,0.000009418352,0.00007248815,0.0007017003,0.0001306363,0.0003739801,0.0001693908,0.0001211243,0.00146612,0.00001092285],"study_design_candidate":"observational","study_design_consensus":"observational","genre_codex":"empirical","genre_gemma":"empirical","genre_scores_codex":[0.9866567,0.0001770011,0.000354797,0.00006000433,0.000007580582,0.00003831924,0.01131825,0.00001655582,0.001370892],"genre_scores_gemma":[0.9826871,0.0001033142,0.0002578661,0.0001062683,0.00001797609,0.00008796089,0.01582901,0.00001078358,0.0008997558],"genre_candidate":"empirical","genre_consensus":"empirical","teacher_disagreement_score":0.005064064,"threshold_uncertainty_score":0.01006919,"prediction_status":"machine_predicted_unvalidated"},"machine_scores":{"provisional":true,"baseline":true,"maturity_gate_passed":false,"score_opus":0.02329628316413295,"score_gpt":0.2508625316525623,"score_spread":0.2275662484884293,"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."}}