{"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":"metacan-v3-hybrid-931329e0061c","candidate_categories":[],"consensus_categories":[],"category_scores_codex":[0.003852984,0.0003345834,0.0007609563,0.0002576642,0.0002494334,0.001386758,0.0003660161,0.002050056,0.004924263],"category_scores_gemma":[0.002066823,0.00008228338,0.0006287415,0.0002952224,0.000567687,0.001104743,0.0004156945,0.002493013,0.0009144986],"about_ca_system_candidate":false,"about_ca_system_consensus":false,"about_ca_system_score_codex":0.0005261212,"about_ca_system_score_gemma":0.0005614951,"about_ca_topic_candidate":false,"about_ca_topic_consensus":false,"about_ca_topic_score_codex":0.0006611588,"about_ca_topic_score_gemma":0.001640772,"domain_scores_codex":[0.9989291,0.0005518938,0.00005305039,0.00008503565,0.0002849282,0.00009605469],"domain_scores_gemma":[0.9988484,0.0007195559,0.0001174883,0.00005217003,0.00008587648,0.0001763858],"domain_codex":null,"domain_gemma":null,"domain_candidate":null,"domain_consensus":null,"study_design_codex":"design_other","study_design_gemma":"not_applicable","study_design_scores_codex":[0.04168499,0.001889331,0.003519791,0.001665419,0.0005533806,0.002085664,0.0003659723,0.003241916,0.03849451,0.03584965,0.04916659,0.8214828],"study_design_scores_gemma":[0.03741385,0.09435652,0.01820218,0.001377148,0.0008801648,0.005246253,0.0003869076,0.005294797,0.01751027,0.02423921,0.7949327,0.0001600054],"study_design_candidate":"not_applicable","study_design_consensus":null,"genre_codex":"empirical","genre_gemma":"commentary","genre_scores_codex":[0.4751239,0.2889983,0.01854441,0.1494127,0.007758233,0.001272606,0.002494989,0.0007804854,0.05561453],"genre_scores_gemma":[0.8119782,0.09774818,0.01522859,0.03543172,0.006531795,0.0007195027,0.002574432,0.0002040057,0.02958361],"genre_candidate":"commentary","genre_consensus":null,"teacher_disagreement_score":0.004924263,"threshold_uncertainty_score":0.02037674,"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."}}