{"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":"codex-gemma-dda1882f352a","candidate_categories":[],"consensus_categories":[],"category_scores_codex":[0.0001211494,0.00009049612,0.0001326607,0.00008512817,0.00006143857,0.000004447401,0.0000921503,0.00006166875,0.00001899615],"category_scores_gemma":[0.0001038028,0.00008282836,0.00001315684,0.0001269648,0.0001202752,0.000002178131,0.0002293651,0.0001745133,5.937592e-7],"about_ca_system_candidate":false,"about_ca_system_consensus":false,"about_ca_system_score_codex":0.000002369004,"about_ca_system_score_gemma":0.00002984185,"about_ca_topic_candidate":false,"about_ca_topic_consensus":false,"about_ca_topic_score_codex":0.00009461781,"about_ca_topic_score_gemma":0.0001022612,"domain_scores_codex":[0.999145,0.0001319471,0.0001307419,0.0003062375,0.0001046025,0.0001815032],"domain_scores_gemma":[0.9996595,0.00004198559,0.00004824617,0.0001716118,0.00002663853,0.00005202142],"domain_codex":null,"domain_gemma":null,"domain_candidate":null,"domain_consensus":null,"study_design_codex":"observational","study_design_gemma":"observational","study_design_scores_codex":[0.009050648,0.0005113965,0.6585305,0.00006608866,0.0001161396,0.00007752094,0.0006684484,0.0008617403,0.3171736,0.000267264,0.001137358,0.0115393],"study_design_scores_gemma":[0.006946134,0.007995354,0.9527948,0.000004697901,0.000008543871,0.000413991,0.00004988694,0.000106805,0.01917544,0.00001559881,0.01241579,0.00007289128],"study_design_candidate":"observational","study_design_consensus":"observational","genre_codex":"empirical","genre_gemma":"empirical","genre_scores_codex":[0.9983925,0.0006280997,0.00001017413,0.0002797405,0.00001513582,0.0003249181,0.00002479813,0.000005331438,0.0003193057],"genre_scores_gemma":[0.9991826,0.0002296767,0.00003987906,0.000330468,0.00002499668,0.00002658773,0.00006010344,0.00001292484,0.00009275271],"genre_candidate":"empirical","genre_consensus":"empirical","teacher_disagreement_score":0.2979982,"threshold_uncertainty_score":0.3377642,"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."}}