{"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":"metacan-v3-hybrid-931329e0061c","candidate_categories":[],"consensus_categories":[],"category_scores_codex":[0.0009052882,0.0005161224,0.0007483934,0.0001549519,0.0002840604,0.0005043494,0.0002435619,0.0008878388,0.002092461],"category_scores_gemma":[0.0007014534,0.0002058095,0.0006274882,0.0001351112,0.0004525134,0.0006188623,0.0001690696,0.001595039,0.0003950679],"about_ca_system_candidate":false,"about_ca_system_consensus":false,"about_ca_system_score_codex":0.0005112901,"about_ca_system_score_gemma":0.0004929836,"about_ca_topic_candidate":false,"about_ca_topic_consensus":false,"about_ca_topic_score_codex":0.0006255046,"about_ca_topic_score_gemma":0.001185353,"domain_scores_codex":[0.999754,0.00009328919,0.00001655651,0.00006109933,0.00003490114,0.00004015894],"domain_scores_gemma":[0.9996492,0.00009274931,0.00008063843,0.00002557948,0.00003291033,0.000118964],"domain_codex":null,"domain_gemma":null,"domain_candidate":null,"domain_consensus":null,"study_design_codex":"randomized_trial","study_design_gemma":"nonrandomized_trial","study_design_scores_codex":[0.5342615,0.03308873,0.005342306,0.001298022,0.0008767897,0.0002752187,0.0005812655,0.002754753,0.1457636,0.0006070154,0.004906662,0.2702441],"study_design_scores_gemma":[0.158988,0.8013809,0.0176641,0.0001432779,0.0005261257,0.0004570419,0.0001523745,0.003062543,0.01237906,0.0004157982,0.004788799,0.00004209212],"study_design_candidate":"nonrandomized_trial","study_design_consensus":null,"genre_codex":"empirical","genre_gemma":"empirical","genre_scores_codex":[0.9953686,0.002188547,0.0003413451,0.0001904491,0.00007032016,0.0003469601,0.000346656,0.00004995042,0.001097165],"genre_scores_gemma":[0.9967443,0.0006825148,0.0005780748,0.0002940673,0.00004160488,0.0004057273,0.0003582754,0.00000919293,0.0008862329],"genre_candidate":"empirical","genre_consensus":"empirical","teacher_disagreement_score":0.002092461,"threshold_uncertainty_score":0.006999969,"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."}}