{"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":"metacan-v3-hybrid-931329e0061c","candidate_categories":[],"consensus_categories":[],"category_scores_codex":[0.0002384342,0.00020907,0.0003798208,0.00009794368,0.0002247935,0.000138194,0.000128289,0.0002560863,0.0003377113],"category_scores_gemma":[0.0005950403,0.00004061859,0.0001648539,0.00006149758,0.0001609228,0.00007745497,0.0001075023,0.0003911624,0.0001198308],"about_ca_system_candidate":false,"about_ca_system_consensus":false,"about_ca_system_score_codex":0.000131338,"about_ca_system_score_gemma":0.00009874511,"about_ca_topic_candidate":false,"about_ca_topic_consensus":false,"about_ca_topic_score_codex":0.0001749324,"about_ca_topic_score_gemma":0.0002829827,"domain_scores_codex":[0.9999201,0.00003346119,0.000009651024,0.00001221575,0.00001340367,0.00001118552],"domain_scores_gemma":[0.9998035,0.0001005862,0.00003061983,0.00002330556,0.000009767905,0.00003229845],"domain_codex":null,"domain_gemma":null,"domain_candidate":null,"domain_consensus":null,"study_design_codex":"design_other","study_design_gemma":"nonrandomized_trial","study_design_scores_codex":[0.01697085,0.00203459,0.1121168,0.000547398,0.0003090187,0.1502376,0.0008864255,0.001155699,0.327225,0.0007814449,0.002193618,0.3855416],"study_design_scores_gemma":[0.001490173,0.05200541,0.2234928,0.0001786112,0.0006309752,0.3938951,0.0005051862,0.003011538,0.2856119,0.0006975679,0.03843114,0.00004970152],"study_design_candidate":"nonrandomized_trial","study_design_consensus":null,"genre_codex":"empirical","genre_gemma":"empirical","genre_scores_codex":[0.988896,0.007498318,0.00172494,0.000279362,0.00005997682,0.00002393374,0.00006552354,0.00002904494,0.001422911],"genre_scores_gemma":[0.9964303,0.002267849,0.0006915725,0.0001102322,0.00003962762,0.00001018687,0.00009089748,0.000005044062,0.0003542942],"genre_candidate":"empirical","genre_consensus":"empirical","teacher_disagreement_score":0.0003798208,"threshold_uncertainty_score":0.001260996,"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."}}