{"id":"W4308558941","doi":"10.1016/j.ymgme.2022.11.002","title":"Venglustat, an orally administered glucosylceramide synthase inhibitor: Assessment over 3 years in adult males with classic Fabry disease in an open-label phase 2 study and its extension study","year":2022,"lang":"en","type":"article","venue":"Molecular Genetics and Metabolism","topic":"Lysosomal Storage Disorders Research","field":"Medicine","cited_by":49,"is_retracted":false,"has_abstract":true,"ca_institutions":"Sanofi (Canada)","funders":"Actelion Pharmaceuticals; University of Minnesota; School of Medicine, Emory University; Emory University; Idorsia Pharmaceuticals; Amicus Therapeutics; Cincinnati Children's Hospital Medical Center; Sangamo Therapeutics; National Institute of Neurological Disorders and Stroke; Sanofi; Alexion Pharmaceuticals; Pfizer","keywords":"Adverse effect; Medicine; Globotriaosylceramide; Fabry disease; Internal medicine; Pharmacodynamics; Pharmacokinetics; Disease; Pharmacology; Gastroenterology","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":[0.002256127,0.0008736336,0.001544859,0.0003329082,0.000277934,0.0004997732,0.0004110785,0.0008466141,0.001329734],"category_scores_gemma":[0.0008647643,0.0003087517,0.00110378,0.0002825115,0.0004239039,0.0005611695,0.0002692611,0.001113526,0.0002871433],"about_ca_system_candidate":false,"about_ca_system_consensus":false,"about_ca_system_score_codex":0.000408318,"about_ca_system_score_gemma":0.0006993278,"about_ca_topic_candidate":false,"about_ca_topic_consensus":false,"about_ca_topic_score_codex":0.0006479541,"about_ca_topic_score_gemma":0.001167941,"domain_scores_codex":[0.9995771,0.0002044244,0.00002716115,0.00006661533,0.00005771265,0.00006702156],"domain_scores_gemma":[0.9991515,0.0001274011,0.0002506032,0.00004186108,0.00007930228,0.0003493169],"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":[0.6592195,0.04394939,0.07415975,0.001079533,0.002770916,0.0009724553,0.0008214205,0.003493257,0.06548952,0.0002181558,0.001888784,0.1459372],"study_design_scores_gemma":[0.1328162,0.7015054,0.1543381,0.00006243109,0.001364781,0.0008705204,0.0001674846,0.001995027,0.003882506,0.0001234077,0.002815341,0.00005873259],"study_design_candidate":"nonrandomized_trial","study_design_consensus":null,"genre_codex":"empirical","genre_gemma":"empirical","genre_scores_codex":[0.9966671,0.001833767,0.0002731897,0.00006912208,0.00001626702,0.0003782054,0.0003609798,0.00002524178,0.0003760158],"genre_scores_gemma":[0.9959611,0.001263341,0.0005342693,0.0002576628,0.00005578465,0.0004267011,0.0008159402,0.000006619548,0.0006784704],"genre_candidate":"empirical","genre_consensus":"empirical","teacher_disagreement_score":0.002256127,"threshold_uncertainty_score":0.01193166,"prediction_status":"machine_predicted_unvalidated"},"machine_scores":{"provisional":true,"baseline":true,"maturity_gate_passed":false,"score_opus":0.03919698044442316,"score_gpt":0.3755322938600135,"score_spread":0.3363353134155904,"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."}}