{"id":"W4400830763","doi":"10.1002/jimd.12771","title":"Renal and multisystem effectiveness of 3.9 years of migalastat in a global real‐world cohort: Results from the <scp>followME</scp> Fabry <scp>Pathfinders</scp> registry","year":2024,"lang":"en","type":"article","venue":"Journal of Inherited Metabolic Disease","topic":"Lysosomal Storage Disorders Research","field":"Medicine","cited_by":18,"is_retracted":false,"has_abstract":true,"ca_institutions":"University of British Columbia; Université de Montréal; Hôpital du Sacré-Cœur de Montréal; Dalhousie University","funders":"Amicus Therapeutics","keywords":"Medicine; Fabry disease; Cohort; Renal function; Internal medicine; Prospective cohort study; 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":[0.002551246,0.000234189,0.0004642584,0.0003265818,0.0002548198,0.000864175,0.0003454214,0.0005565492,0.0009403649],"category_scores_gemma":[0.002939908,0.0001226522,0.001168823,0.0005240061,0.0002683951,0.0006202825,0.0005727576,0.000615266,0.0001506302],"about_ca_system_candidate":false,"about_ca_system_consensus":false,"about_ca_system_score_codex":0.0006053273,"about_ca_system_score_gemma":0.0004265759,"about_ca_topic_candidate":false,"about_ca_topic_consensus":false,"about_ca_topic_score_codex":0.003912654,"about_ca_topic_score_gemma":0.004872429,"domain_scores_codex":[0.998858,0.0005344974,0.00008119689,0.000247148,0.0001364079,0.0001426947],"domain_scores_gemma":[0.9973674,0.0005815883,0.001181641,0.0002266565,0.0001212268,0.0005214664],"domain_codex":null,"domain_gemma":null,"domain_candidate":null,"domain_consensus":null,"study_design_codex":"observational","study_design_gemma":"observational","study_design_scores_codex":[0.008230254,0.0005447346,0.9785197,0.00004337998,0.0008049661,0.0002007235,0.0001154562,0.001394982,0.0006095801,0.00005413079,0.0006096464,0.008872431],"study_design_scores_gemma":[0.0003912023,0.004315606,0.991904,0.00001568265,0.0004524405,0.0002573715,0.0001817389,0.001592097,0.0002484357,0.00006405195,0.0005585563,0.00001885588],"study_design_candidate":"observational","study_design_consensus":"observational","genre_codex":"empirical","genre_gemma":"empirical","genre_scores_codex":[0.9989063,0.0003462259,0.00004343217,0.00006422832,0.000005864338,0.000005405677,0.000453653,0.000004572952,0.0001703749],"genre_scores_gemma":[0.9991917,0.0001038753,0.00003054703,0.00004611289,0.000009850142,0.000005326711,0.0005374709,0.000002010654,0.00007301044],"genre_candidate":"empirical","genre_consensus":"empirical","teacher_disagreement_score":0.003912654,"threshold_uncertainty_score":0.01349241,"prediction_status":"machine_predicted_unvalidated"},"machine_scores":{"provisional":true,"baseline":true,"maturity_gate_passed":false,"score_opus":0.0148360740143923,"score_gpt":0.2937716054044788,"score_spread":0.2789355313900865,"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."}}