{"id":"W1975365119","doi":"10.1086/318809","title":"A Phase 1/2 Clinical Trial of Enzyme Replacement in Fabry Disease: Pharmacokinetic, Substrate Clearance, and Safety Studies","year":2001,"lang":"en","type":"article","venue":"The American Journal of Human Genetics","topic":"Lysosomal Storage Disorders Research","field":"Medicine","cited_by":383,"is_retracted":false,"has_abstract":false,"ca_institutions":"","funders":"Eunice Kennedy Shriver National Institute of Child Health and Human Development; National Institute of Diabetes and Digestive and Kidney Diseases; National Center for Research Resources; McGill University","keywords":"Pharmacokinetics; Medicine; Enzyme replacement therapy; Globotriaosylceramide; Kidney; Fabry disease; Internal medicine; Clearance; Pharmacology; Endocrinology; Pathology; Gastroenterology; Urology; Disease","routes":{"ca_aff":false,"ca_fund":true,"ca_venue":false,"about_ca":false,"invisible_to_affiliation_only":true},"retraction":null,"screen":null,"direct_labels":[],"prediction":{"model_version":"metacan-v3-hybrid-931329e0061c","candidate_categories":[],"consensus_categories":[],"category_scores_codex":[0.002965919,0.003286725,0.003518159,0.0007373173,0.0006475782,0.001351341,0.0009807266,0.003264067,0.003379894],"category_scores_gemma":[0.002094242,0.0007016483,0.002120923,0.0004910472,0.001682931,0.001540018,0.000305353,0.004476864,0.0009454412],"about_ca_system_candidate":false,"about_ca_system_consensus":false,"about_ca_system_score_codex":0.00108857,"about_ca_system_score_gemma":0.001460837,"about_ca_topic_candidate":false,"about_ca_topic_consensus":false,"about_ca_topic_score_codex":0.000998117,"about_ca_topic_score_gemma":0.001150129,"domain_scores_codex":[0.9991335,0.000454776,0.00003338558,0.0001382848,0.00008551878,0.0001545459],"domain_scores_gemma":[0.9985044,0.0004280149,0.0002060093,0.0001756297,0.00009705896,0.0005889532],"domain_codex":null,"domain_gemma":null,"domain_candidate":null,"domain_consensus":null,"study_design_codex":"randomized_trial","study_design_gemma":"nonrandomized_trial","study_design_scores_codex":[0.8376684,0.04326308,0.00284968,0.0004854029,0.001206733,0.0005714065,0.0003464527,0.002663919,0.03632041,0.0005278341,0.005345337,0.06875139],"study_design_scores_gemma":[0.6215625,0.3638261,0.005237591,0.0000288671,0.0003382183,0.0006548016,0.00004621659,0.001347924,0.004377398,0.0004383896,0.002094407,0.00004760869],"study_design_candidate":"nonrandomized_trial","study_design_consensus":null,"genre_codex":"empirical","genre_gemma":"empirical","genre_scores_codex":[0.9887481,0.003627863,0.001840803,0.001038761,0.0002156099,0.001361662,0.001181692,0.0001957726,0.001789779],"genre_scores_gemma":[0.9883659,0.002338627,0.002185475,0.001296169,0.0003199405,0.001333536,0.001668308,0.00009176647,0.002400377],"genre_candidate":"empirical","genre_consensus":"empirical","teacher_disagreement_score":0.003518159,"threshold_uncertainty_score":0.01568544,"prediction_status":"machine_predicted_unvalidated"},"machine_scores":{"provisional":true,"baseline":true,"maturity_gate_passed":false,"score_opus":0.1037982530427376,"score_gpt":0.4664230837248811,"score_spread":0.3626248306821435,"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."}}