{"id":"W4406043541","doi":"10.1002/ctd2.70028","title":"The FACTs trial for Fabry disease highlights the promise and challenges of gene therapy","year":2025,"lang":"en","type":"article","venue":"Clinical and Translational Discovery","topic":"Lysosomal Storage Disorders Research","field":"Medicine","cited_by":0,"is_retracted":false,"has_abstract":true,"ca_institutions":"Dalhousie University","funders":"","keywords":"Fabry disease; Genetic enhancement; Disease; Medicine; Clinical trial; Intensive care medicine; Genetics; Gene; Biology; Internal medicine","routes":{"ca_aff":true,"ca_fund":false,"ca_venue":false,"about_ca":true,"invisible_to_affiliation_only":false},"retraction":null,"screen":null,"direct_labels":[],"prediction":{"model_version":"codex-gemma-dda1882f352a","candidate_categories":[],"consensus_categories":[],"category_scores_codex":[0.000430912,0.00008195048,0.0001920896,0.00002059905,0.0001741438,0.00003071872,0.00007472697,0.00005666168,0.000003539527],"category_scores_gemma":[0.0002619787,0.00003764466,0.0001430585,0.00005021822,0.0004768495,0.00007272951,0.00001359014,0.0001046311,3.354557e-7],"about_ca_system_candidate":false,"about_ca_system_consensus":false,"about_ca_system_score_codex":0.000002022827,"about_ca_system_score_gemma":0.000201602,"about_ca_topic_candidate":false,"about_ca_topic_consensus":false,"about_ca_topic_score_codex":0.000003790037,"about_ca_topic_score_gemma":0.00001051467,"domain_scores_codex":[0.9990925,0.00008650473,0.0003100121,0.0002011482,0.0001949448,0.0001148631],"domain_scores_gemma":[0.9976765,0.001993561,0.0000397044,0.0001479034,0.00006678521,0.00007556661],"domain_codex":null,"domain_gemma":null,"domain_candidate":null,"domain_consensus":null,"study_design_codex":"design_other","study_design_gemma":"observational","study_design_scores_codex":[0.2603504,0.001795645,0.03799624,0.0006301404,0.001012861,0.000008489918,0.0004492935,0.000004661151,0.0001414645,0.2654788,0.0005442554,0.4315877],"study_design_scores_gemma":[0.05633043,0.0006523511,0.8603376,0.00008121313,0.0001314127,9.373519e-7,0.00009296975,0.0003049508,0.00009942734,0.05426819,0.02760692,0.00009364959],"study_design_candidate":"observational","study_design_consensus":null,"genre_codex":"empirical","genre_gemma":"empirical","genre_scores_codex":[0.8498764,0.05781745,0.0004622702,0.08940873,0.0001699378,0.001768806,0.00006621623,0.000008244738,0.0004219524],"genre_scores_gemma":[0.9724818,0.02643581,0.00003351926,0.0002094207,0.0001558159,0.00009969312,0.00001723034,0.00000594317,0.0005607895],"genre_candidate":"empirical","genre_consensus":"empirical","teacher_disagreement_score":0.8223413,"threshold_uncertainty_score":0.1756972,"prediction_status":"machine_predicted_unvalidated"},"machine_scores":{"provisional":true,"baseline":true,"maturity_gate_passed":false,"score_opus":0.1132710562655968,"score_gpt":0.3922275654434135,"score_spread":0.2789565091778167,"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."}}