{"id":"W4379967447","doi":"10.1038/s41591-023-02398-1","title":"Trofinetide for the treatment of Rett syndrome: a randomized phase 3 study","year":2023,"lang":"en","type":"article","venue":"Nature Medicine","topic":"Genetics and Neurodevelopmental Disorders","field":"Biochemistry, Genetics and Molecular Biology","cited_by":186,"is_retracted":false,"has_abstract":true,"ca_institutions":"","funders":"National Institute of Neurological Disorders and Stroke; University of California, San Diego; University of North Carolina at Chapel Hill; National Institutes of Health; Eunice Kennedy Shriver National Institute of Child Health and Human Development; AveXis; Zynerba Pharmaceuticals; National Institute of Child Health and Human Development; Children's Hospital Colorado; GW Pharmaceuticals; Ionis Pharmaceuticals; International Foundation for CDKL5 Research; Texas Children's Hospital; Ultragenyx Pharmaceutical; Zogenix; MIND Institute, University of California, Davis; ACADIA Pharmaceuticals; University of South Florida; University of Cincinnati; Rush University; LouLou Foundation; Clinical Trial Center, China Medical University Hospital; Mallinckrodt Pharmaceuticals; University of California, Davis; Cincinnati Children's Hospital Medical Center; Emory University; Cleveland Clinic; University of Pennsylvania; Vanderbilt University; Vanderbilt University Medical Center; Marinus Pharmaceuticals; Children's Hospital of Philadelphia; Biogen; International Rett Syndrome Foundation; Simons Foundation","keywords":"Rett syndrome; Placebo; Medicine; Internal medicine; Clinical endpoint; Pediatrics; Clinical trial; Pathology; Biology","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":[0.0003007664,0.0001311421,0.0003099319,0.000051548,0.00005615938,0.000002442771,0.0001265168,0.0000892867,0.0000144682],"category_scores_gemma":[0.0002184961,0.00006885094,0.0001068498,0.0001694051,0.0001143258,4.676107e-7,0.00002005571,0.00005319353,0.000001599195],"about_ca_system_candidate":false,"about_ca_system_consensus":false,"about_ca_system_score_codex":0.000005246977,"about_ca_system_score_gemma":0.0000245989,"about_ca_topic_candidate":false,"about_ca_topic_consensus":false,"about_ca_topic_score_codex":0.00001150523,"about_ca_topic_score_gemma":0.00001683478,"domain_scores_codex":[0.9992146,0.00005123655,0.0002224349,0.0002191114,0.0001480887,0.0001444813],"domain_scores_gemma":[0.9994543,0.0001310479,0.000069945,0.000254422,0.00005507715,0.00003520036],"domain_codex":null,"domain_gemma":null,"domain_candidate":null,"domain_consensus":null,"study_design_codex":"bench_or_experimental","study_design_gemma":"randomized_trial","study_design_scores_codex":[0.06290506,0.002741112,0.001187605,0.00009777559,0.002110211,0.0000563428,0.00274819,0.0000894769,0.8495032,0.0001349365,0.04218662,0.03623952],"study_design_scores_gemma":[0.7006742,0.02226469,0.02054501,0.00004336978,0.0008175398,0.00003076329,0.002200943,0.0005099377,0.09484091,0.0002586007,0.1574399,0.0003741382],"study_design_candidate":"bench_or_experimental","study_design_consensus":null,"genre_codex":"empirical","genre_gemma":"empirical","genre_scores_codex":[0.9908962,0.005023018,0.0001293655,0.001733389,0.0003597874,0.001720063,0.00001818971,0.00001031288,0.0001096534],"genre_scores_gemma":[0.995717,0.002249721,0.00005593854,0.0002162626,0.0000588904,0.0002716561,0.000154797,0.00001671166,0.001258946],"genre_candidate":"empirical","genre_consensus":"empirical","teacher_disagreement_score":0.7546622,"threshold_uncertainty_score":0.280766,"prediction_status":"machine_predicted_unvalidated"},"machine_scores":{"provisional":true,"baseline":true,"maturity_gate_passed":false,"score_opus":0.01233716112357668,"score_gpt":0.3183866605019933,"score_spread":0.3060494993784166,"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."}}