{"id":"W3047518349","doi":"10.1016/j.pediatrneurol.2020.08.001","title":"A Phase 2 Study of AMO-02 (Tideglusib) in Congenital and Childhood-Onset Myotonic Dystrophy Type 1 (DM1)","year":2020,"lang":"en","type":"article","venue":"Pediatric Neurology","topic":"Genetic Neurodegenerative Diseases","field":"Neuroscience","cited_by":89,"is_retracted":false,"has_abstract":true,"ca_institutions":"Ottawa Hospital; Children's Hospital of Eastern Ontario; University of Ottawa","funders":"Canadian Institutes of Health Research; Canada Research Chairs; AMO Pharma; Canada Foundation for Innovation","keywords":"Myotonic dystrophy; Medicine; Tolerability; Area under the curve; Placebo; Internal medicine; Adverse effect; Anesthesia; Pediatrics; Pathology","routes":{"ca_aff":true,"ca_fund":true,"ca_venue":false,"about_ca":false,"invisible_to_affiliation_only":false},"retraction":null,"screen":null,"direct_labels":[],"prediction":{"model_version":"codex-gemma-dda1882f352a","candidate_categories":[],"consensus_categories":[],"category_scores_codex":[0.00006187252,0.000241267,0.0003787482,0.0002161197,0.00006276398,0.00001893811,0.0003196334,0.00006110266,0.00006504665],"category_scores_gemma":[0.001095166,0.0002427991,0.00004580355,0.0008139947,0.0001362186,0.00007102501,0.000269303,0.0003157427,0.00003690892],"about_ca_system_candidate":false,"about_ca_system_consensus":false,"about_ca_system_score_codex":0.00000477973,"about_ca_system_score_gemma":0.0001071051,"about_ca_topic_candidate":false,"about_ca_topic_consensus":false,"about_ca_topic_score_codex":0.00002112859,"about_ca_topic_score_gemma":0.00001221843,"domain_scores_codex":[0.9975534,0.000566251,0.0004348525,0.0008378449,0.0002462722,0.0003613526],"domain_scores_gemma":[0.9989254,0.0003650321,0.000165121,0.0002807279,0.00004609402,0.0002175873],"domain_codex":null,"domain_gemma":null,"domain_candidate":null,"domain_consensus":null,"study_design_codex":"observational","study_design_gemma":"observational","study_design_scores_codex":[0.002609956,0.01022366,0.8099536,0.00009850672,0.00002711581,0.001758649,0.005126727,0.0005891809,0.1675061,0.00008757573,0.00133542,0.0006835433],"study_design_scores_gemma":[0.02464334,0.04815656,0.8554378,0.000001924976,0.0003991845,0.0002027232,0.0002852279,0.002065087,0.0674147,0.0002084934,0.0002633384,0.0009216236],"study_design_candidate":"observational","study_design_consensus":"observational","genre_codex":"empirical","genre_gemma":"empirical","genre_scores_codex":[0.9981253,0.0001855139,0.000002985238,0.0004806173,0.0002396091,0.00078559,0.00006272747,0.0000474024,0.00007032898],"genre_scores_gemma":[0.9974201,0.000133033,0.000009991593,0.002198927,0.0001620672,0.00003405516,0.000002508902,0.00003232748,0.000007044088],"genre_candidate":"empirical","genre_consensus":"empirical","teacher_disagreement_score":0.1000914,"threshold_uncertainty_score":0.990106,"prediction_status":"machine_predicted_unvalidated"},"machine_scores":{"provisional":true,"baseline":true,"maturity_gate_passed":false,"score_opus":0.0251983148847592,"score_gpt":0.2737161198047742,"score_spread":0.248517804920015,"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."}}