{"id":"W4308686111","doi":"10.1056/nejmoa2200587","title":"In Utero Enzyme-Replacement Therapy for Infantile-Onset Pompe’s Disease","year":2022,"lang":"en","type":"article","venue":"New England Journal of Medicine","topic":"Lysosomal Storage Disorders Research","field":"Medicine","cited_by":120,"is_retracted":false,"has_abstract":true,"ca_institutions":"University of Ottawa; Ottawa Hospital; Children's Hospital of Eastern Ontario","funders":"Eunice Kennedy Shriver National Institute of Child Health and Human Development; National Institute of Diabetes and Digestive and Kidney Diseases; University of California, San Francisco; National Institutes of Health; Sanofi; CHEO Research Institute; Sanofi Genzyme","keywords":"Enzyme replacement therapy; In utero; Disease; Medicine; Glycogen storage disease type II; Cardiomyopathy; Fetus; Pediatrics; Biomarker; Pregnancy; Internal medicine; Heart failure; Biology","routes":{"ca_aff":true,"ca_fund":true,"ca_venue":false,"about_ca":false,"invisible_to_affiliation_only":false},"retraction":null,"screen":null,"direct_labels":[],"prediction":{"model_version":"codex-gemma-dda1882f352a","candidate_categories":["insufficient_payload"],"consensus_categories":[],"category_scores_codex":[0.001611194,0.0001499109,0.0004843048,0.0004618516,0.00009153448,0.000008087283,0.0002136955,0.00002679275,0.002452207],"category_scores_gemma":[0.0006185886,0.0001038899,0.00011449,0.0003039692,0.00006880108,0.00008825077,0.00005555366,0.0005185381,0.000002046963],"about_ca_system_candidate":false,"about_ca_system_consensus":false,"about_ca_system_score_codex":0.000187027,"about_ca_system_score_gemma":0.0005614859,"about_ca_topic_candidate":false,"about_ca_topic_consensus":false,"about_ca_topic_score_codex":0.0001403587,"about_ca_topic_score_gemma":0.00001851666,"domain_scores_codex":[0.9977343,0.0001653473,0.0005800471,0.0001719663,0.001036998,0.0003113071],"domain_scores_gemma":[0.9986248,0.0002907272,0.0002129335,0.0002212021,0.0001580729,0.0004922586],"domain_codex":null,"domain_gemma":null,"domain_candidate":null,"domain_consensus":null,"study_design_codex":"not_applicable","study_design_gemma":"not_applicable","study_design_scores_codex":[0.1244226,0.001801791,0.1807075,0.0002588493,0.0004537322,0.006856026,0.00714804,0.0004657637,0.004222901,0.0002540388,0.5046962,0.1687127],"study_design_scores_gemma":[0.2048068,0.01047131,0.02214567,0.00020204,0.00009503244,0.002117927,0.0007737532,0.0003802125,0.000278063,0.001151354,0.7574185,0.0001594167],"study_design_candidate":"not_applicable","study_design_consensus":"not_applicable","genre_codex":"empirical","genre_gemma":"empirical","genre_scores_codex":[0.9342655,0.006799187,0.0005051265,0.05663889,0.0007436093,0.0007485407,0.00001752071,0.000009217238,0.0002724358],"genre_scores_gemma":[0.9910011,0.0007205181,0.0001993428,0.004205199,0.001878859,0.0000333618,0.0000267181,0.00002716624,0.00190772],"genre_candidate":"empirical","genre_consensus":"empirical","teacher_disagreement_score":0.2527223,"threshold_uncertainty_score":0.9984597,"prediction_status":"machine_predicted_unvalidated"},"machine_scores":{"provisional":true,"baseline":true,"maturity_gate_passed":false,"score_opus":0.02719519212244257,"score_gpt":0.3347295823122702,"score_spread":0.3075343901898276,"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."}}