{"id":"W4408318778","doi":"10.1002/jimd.70014","title":"Safety and Efficacy of <scp>DTX401</scp> , an <scp>AAV8</scp> ‐Mediated Liver‐Directed Gene Therapy, in Adults With Glycogen Storage Disease Type I a ( <scp>GSDIa</scp> )","year":2025,"lang":"en","type":"article","venue":"Journal of Inherited Metabolic Disease","topic":"Glycogen Storage Diseases and Myoclonus","field":"Medicine","cited_by":21,"is_retracted":false,"has_abstract":true,"ca_institutions":"Montreal Children's Hospital","funders":"Moderna; McGill University Health Centre; Danone; Vitaflo; McGill University; Ultragenyx Pharmaceutical; Sanofi","keywords":"Discontinuation; Medicine; Internal medicine; Glycogen storage disease type I; Glycogen storage disease; Adverse effect; Hypoglycemia; Gastroenterology; Pharmacology; Glycogen; Endocrinology; Insulin","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":["metaepi_narrow"],"consensus_categories":[],"category_scores_codex":[0.0007398012,0.0009121756,0.001952468,0.001418632,0.000189472,0.0001145167,0.0005854339,0.0002938572,0.00004251796],"category_scores_gemma":[0.00620037,0.0007324103,0.0005870917,0.002749162,0.0004013212,0.0005727015,0.0001679413,0.0008573113,0.00001323196],"about_ca_system_candidate":false,"about_ca_system_consensus":false,"about_ca_system_score_codex":0.0001565148,"about_ca_system_score_gemma":0.00228646,"about_ca_topic_candidate":false,"about_ca_topic_consensus":false,"about_ca_topic_score_codex":0.0001768999,"about_ca_topic_score_gemma":0.00003321461,"domain_scores_codex":[0.9943903,0.0006285459,0.001738771,0.0009049398,0.001295762,0.001041693],"domain_scores_gemma":[0.9917377,0.001070368,0.001226063,0.00105352,0.001703232,0.003209081],"domain_codex":null,"domain_gemma":null,"domain_candidate":null,"domain_consensus":null,"study_design_codex":"observational","study_design_gemma":"observational","study_design_scores_codex":[0.07966372,0.05167057,0.506515,0.007216417,0.0153033,0.1145943,0.02198718,0.002569519,0.09006413,0.0009399038,0.02445958,0.08501638],"study_design_scores_gemma":[0.02790633,0.001645761,0.9132962,0.001782616,0.003092385,0.0002409023,0.001645805,0.001374077,0.0009428191,0.00006088741,0.04784112,0.0001710817],"study_design_candidate":"observational","study_design_consensus":"observational","genre_codex":"empirical","genre_gemma":"empirical","genre_scores_codex":[0.9082878,0.0884692,0.00007313793,0.0001236897,0.0004229832,0.001541018,0.0006177145,0.0001388956,0.000325572],"genre_scores_gemma":[0.9736495,0.02222992,0.0007458334,0.001343645,0.0005354219,0.00004761449,0.00043215,0.0001696562,0.0008462642],"genre_candidate":"empirical","genre_consensus":"empirical","teacher_disagreement_score":0.4067812,"threshold_uncertainty_score":0.9995127,"prediction_status":"machine_predicted_unvalidated"},"machine_scores":{"provisional":true,"baseline":true,"maturity_gate_passed":false,"score_opus":0.009598642025679659,"score_gpt":0.2488538337936804,"score_spread":0.2392551917680008,"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."}}