{"id":"W4210359797","doi":"10.1212/nxg.0000000000000657","title":"Therapy Trial Design in Vanishing White Matter","year":2022,"lang":"en","type":"review","venue":"Neurology Genetics","topic":"RNA regulation and disease","field":"Biochemistry, Genetics and Molecular Biology","cited_by":32,"is_retracted":false,"has_abstract":true,"ca_institutions":"","funders":"National Institute of Neurological Disorders and Stroke; National Institutes of Health; Amsterdam Neuroscience; Association Européenne contre les Leucodystrophies; National Center for Advancing Translational Sciences; Nederlandse Organisatie voor Wetenschappelijk Onderzoek; Ministero della Salute; Vrije Universiteit Amsterdam; Hersenstichting; Denali Therapeutics; ZonMw; bluebird bio; McGill University; McGill University Health Centre; Stichting Metakids; Biogen; Fonds de Recherche du Québec - Santé; BC Children's Hospital; Eli Lilly and Company","keywords":"Disease; Medicine; White matter; Leukodystrophy; Clinical trial; Pediatrics; Internal medicine; Magnetic resonance imaging","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":["metaepi_narrow"],"consensus_categories":[],"category_scores_codex":[0.0002479948,0.0003381299,0.0005975286,0.0001337654,0.00006395743,0.00003016128,0.0004291376,0.000479077,0.0007882843],"category_scores_gemma":[0.00003689289,0.0003252872,0.0002974636,0.0001477397,0.00005913426,0.000001470753,0.0001949214,0.0003566213,0.00004912107],"about_ca_system_candidate":false,"about_ca_system_consensus":false,"about_ca_system_score_codex":0.00001188987,"about_ca_system_score_gemma":0.0002702315,"about_ca_topic_candidate":false,"about_ca_topic_consensus":false,"about_ca_topic_score_codex":0.000001141114,"about_ca_topic_score_gemma":0.000002734656,"domain_scores_codex":[0.9974294,0.001013481,0.0004797573,0.0006200126,0.0001443016,0.0003130754],"domain_scores_gemma":[0.9990304,0.0000569953,0.0002162353,0.0005932213,0.00001457112,0.00008860908],"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":[0.01204539,0.000570879,0.001866389,0.0008089197,0.0002069137,0.0001706813,0.00004220177,0.001242497,0.00007469698,0.0000119659,0.02538167,0.9575778],"study_design_scores_gemma":[0.004607948,0.0008502193,0.0001527878,0.00001688256,0.00005764777,0.00003532312,0.000001056046,0.0000146274,0.000004135336,0.00002077524,0.9939725,0.0002660794],"study_design_candidate":"not_applicable","study_design_consensus":null,"genre_codex":"review","genre_gemma":"review","genre_scores_codex":[0.001430233,0.9959421,0.0001606963,0.00007704395,0.000703162,0.001122879,0.00002169004,0.00001169861,0.0005305351],"genre_scores_gemma":[0.00009944534,0.9962322,0.0001620666,0.001979823,0.0003841906,0.0002729368,0.0002784352,0.00008618511,0.0005046951],"genre_candidate":"review","genre_consensus":"review","teacher_disagreement_score":0.9685909,"threshold_uncertainty_score":0.9999199,"prediction_status":"machine_predicted_unvalidated"},"machine_scores":{"provisional":true,"baseline":true,"maturity_gate_passed":false,"score_opus":0.06685039888778331,"score_gpt":0.3224467204340817,"score_spread":0.2555963215462984,"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."}}