{"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":"metacan-v3-hybrid-931329e0061c","candidate_categories":["metaresearch"],"consensus_categories":[],"category_scores_codex":[0.007603178,0.0007184412,0.002309407,0.0004407806,0.0004728893,0.001431271,0.0007633665,0.002584575,0.02384093],"category_scores_gemma":[0.009996993,0.0001963481,0.002669344,0.0004247781,0.0006419993,0.001548078,0.001060918,0.003213235,0.002416899],"about_ca_system_candidate":false,"about_ca_system_consensus":false,"about_ca_system_score_codex":0.000830278,"about_ca_system_score_gemma":0.001634667,"about_ca_topic_candidate":false,"about_ca_topic_consensus":false,"about_ca_topic_score_codex":0.0002431788,"about_ca_topic_score_gemma":0.0006300852,"domain_scores_codex":[0.9937713,0.00477475,0.000404504,0.0004935428,0.0002005848,0.0003554894],"domain_scores_gemma":[0.9961426,0.001933791,0.0005935715,0.0003262178,0.000338136,0.0006657408],"domain_codex":null,"domain_gemma":"methods","domain_candidate":"methods","domain_consensus":null,"study_design_codex":"randomized_trial","study_design_gemma":"not_applicable","study_design_scores_codex":[0.5407676,0.003125424,0.003855006,0.01553532,0.007208532,0.0004681147,0.0003223976,0.003040118,0.00263801,0.01791407,0.04255038,0.3625751],"study_design_scores_gemma":[0.6050252,0.08726714,0.009548239,0.006427537,0.005422136,0.0004742988,0.0002673051,0.006748301,0.001949829,0.04011067,0.2366298,0.0001295309],"study_design_candidate":"not_applicable","study_design_consensus":null,"genre_codex":"empirical","genre_gemma":"review","genre_scores_codex":[0.3343946,0.1785351,0.06070856,0.09933548,0.04661123,0.1105569,0.01794509,0.001888358,0.1500246],"genre_scores_gemma":[0.7138963,0.01749701,0.02900579,0.04444211,0.005659552,0.1554475,0.005515852,0.0002747058,0.02826124],"genre_candidate":"review","genre_consensus":null,"teacher_disagreement_score":0.9923968,"threshold_uncertainty_score":0.07975584,"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."}}