{"id":"W1989476643","doi":"10.1016/j.ymgme.2014.12.179","title":"ENGAGE — A phase 3, randomized, double-blind, placebo-controlled, multi-center study to investigate the efficacy and safety of eliglustat in adults with Gaucher disease type 1: Results after 18months","year":2015,"lang":"en","type":"article","venue":"Molecular Genetics and Metabolism","topic":"Lysosomal Storage Disorders Research","field":"Medicine","cited_by":11,"is_retracted":false,"has_abstract":false,"ca_institutions":"McGill University; Jewish General Hospital; Mount Sinai Hospital","funders":"","keywords":"Double blind; Placebo; Medicine; Disease; Phase (matter); Internal medicine; Physics; Pathology","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":[0.001527118,0.002213452,0.003897775,0.0005171802,0.0007854597,0.001174699,0.001030453,0.002264883,0.006727516],"category_scores_gemma":[0.001367398,0.0007315742,0.002587378,0.000424522,0.001450794,0.001703846,0.0006149134,0.004297788,0.001353251],"about_ca_system_candidate":false,"about_ca_system_consensus":false,"about_ca_system_score_codex":0.0007261622,"about_ca_system_score_gemma":0.001094486,"about_ca_topic_candidate":false,"about_ca_topic_consensus":false,"about_ca_topic_score_codex":0.0009709723,"about_ca_topic_score_gemma":0.002554486,"domain_scores_codex":[0.9994766,0.0002165385,0.00003989528,0.0001126619,0.00005021888,0.0001041547],"domain_scores_gemma":[0.9990169,0.0002310248,0.000239932,0.0001029918,0.00007441484,0.0003346914],"domain_codex":null,"domain_gemma":null,"domain_candidate":null,"domain_consensus":null,"study_design_codex":"randomized_trial","study_design_gemma":"randomized_trial","study_design_scores_codex":[0.9716228,0.009893948,0.0008213248,0.0004905112,0.001119723,0.00004401344,0.00006309855,0.0002396727,0.003528597,0.00007951098,0.0008206455,0.01127614],"study_design_scores_gemma":[0.9311473,0.06545839,0.001856565,0.00002008842,0.0004214036,0.00002791929,0.00002968013,0.0001636107,0.0003749522,0.00009782975,0.0003867161,0.0000155526],"study_design_candidate":"randomized_trial","study_design_consensus":"randomized_trial","genre_codex":"empirical","genre_gemma":"empirical","genre_scores_codex":[0.9858415,0.003436953,0.001164846,0.0007339882,0.0004084406,0.004249755,0.001947436,0.0003778123,0.001839297],"genre_scores_gemma":[0.9799182,0.002247233,0.002394144,0.002357664,0.0005510224,0.006584856,0.002614375,0.0000884839,0.003243941],"genre_candidate":"empirical","genre_consensus":"empirical","teacher_disagreement_score":0.006727516,"threshold_uncertainty_score":0.02250576,"prediction_status":"machine_predicted_unvalidated"},"machine_scores":{"provisional":true,"baseline":true,"maturity_gate_passed":false,"score_opus":0.03046407736822823,"score_gpt":0.3215776516543612,"score_spread":0.291113574286133,"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."}}