{"id":"W4310593690","doi":"10.1212/wnl.0000000000201660","title":"Efficacy and Safety of N-Acetyl- <scp>l</scp> -Leucine in Children and Adults With GM2 Gangliosidoses","year":2022,"lang":"en","type":"article","venue":"Neurology","topic":"Glycosylation and Glycoproteins Research","field":"Biochemistry, Genetics and Molecular Biology","cited_by":35,"is_retracted":false,"has_abstract":true,"ca_institutions":"","funders":"National Institute for Health and Care Research; University of California, Los Angeles; York University","keywords":"Medicine; Sandhoff disease; Pediatrics; Ataxia; Internal medicine; Quality of life (healthcare); Clinical endpoint; Clinical trial; Gastroenterology; Surgery; Disease; Psychiatry","routes":{"ca_aff":false,"ca_fund":true,"ca_venue":false,"about_ca":false,"invisible_to_affiliation_only":true},"retraction":null,"screen":null,"direct_labels":[],"prediction":{"model_version":"metacan-v3-hybrid-931329e0061c","candidate_categories":[],"consensus_categories":[],"category_scores_codex":[0.0009281681,0.0005277193,0.0009146176,0.0001782052,0.0002605715,0.0003668411,0.0003213958,0.0006504231,0.001042153],"category_scores_gemma":[0.001183524,0.0002062075,0.0004847381,0.0001628179,0.0004373465,0.0003805649,0.0002749743,0.0009864818,0.0002642537],"about_ca_system_candidate":false,"about_ca_system_consensus":false,"about_ca_system_score_codex":0.0006076214,"about_ca_system_score_gemma":0.0009625225,"about_ca_topic_candidate":false,"about_ca_topic_consensus":false,"about_ca_topic_score_codex":0.002078114,"about_ca_topic_score_gemma":0.003568099,"domain_scores_codex":[0.9995825,0.0002109428,0.00004301434,0.00005170919,0.00005472434,0.00005721994],"domain_scores_gemma":[0.9989122,0.0001771703,0.000326171,0.00004195864,0.00009307537,0.0004493666],"domain_codex":null,"domain_gemma":null,"domain_candidate":null,"domain_consensus":null,"study_design_codex":"randomized_trial","study_design_gemma":"observational","study_design_scores_codex":[0.7474226,0.02461179,0.08683825,0.000797132,0.0008759393,0.0004034332,0.000674976,0.00116746,0.01898387,0.00009344947,0.001321468,0.1168097],"study_design_scores_gemma":[0.1230429,0.6391483,0.2271814,0.0001640707,0.0005523252,0.0007386529,0.0004092731,0.001247544,0.004772706,0.0000635425,0.002635801,0.00004333429],"study_design_candidate":"observational","study_design_consensus":null,"genre_codex":"empirical","genre_gemma":"empirical","genre_scores_codex":[0.9983702,0.000893558,0.00003871549,0.00004829257,0.000009133219,0.0001164534,0.0001559598,0.00001190761,0.0003557918],"genre_scores_gemma":[0.9980741,0.0006431264,0.0002267579,0.0001236762,0.00002434013,0.0002304905,0.0003398726,0.000003722726,0.0003340339],"genre_candidate":"empirical","genre_consensus":"empirical","teacher_disagreement_score":0.002078114,"threshold_uncertainty_score":0.004908681,"prediction_status":"machine_predicted_unvalidated"},"machine_scores":{"provisional":true,"baseline":true,"maturity_gate_passed":false,"score_opus":0.004244894165954616,"score_gpt":0.2199444386266063,"score_spread":0.2156995444606517,"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."}}