{"id":"W2952212183","doi":"10.1183/23120541.00082-2019","title":"Clinical development of triple-combination CFTR modulators for cystic fibrosis patients with one or two<i>F508del</i>alleles","year":2019,"lang":"en","type":"article","venue":"ERJ Open Research","topic":"Cystic Fibrosis Research Advances","field":"Medicine","cited_by":103,"is_retracted":false,"has_abstract":true,"ca_institutions":"St. Michael's Hospital","funders":"ProQR Therapeutics; Einstein Stiftung Berlin; Bundesministerium für Bildung und Forschung; Arrowhead Pharmaceuticals; Vertex Pharmaceuticals; Genentech; AstraZeneca; PTC Therapeutics; National Institute of Diabetes and Digestive and Kidney Diseases; National Heart, Lung, and Blood Institute; Gilead Sciences","keywords":"Cystic fibrosis; Cystic fibrosis transmembrane conductance regulator; Medicine; Allele; Regulator; Gene; Mutation; Transmembrane protein; Ivacaftor; Genetics; Internal medicine; Receptor; Biology","routes":{"ca_aff":true,"ca_fund":false,"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":[0.004192594,0.0002014617,0.0007606502,0.0003822607,0.0002155506,0.0001083106,0.0007647037,0.0001096196,0.0005594303],"category_scores_gemma":[0.003345933,0.0001402241,0.00008853556,0.0008306772,0.0002784074,0.0003424585,0.0006195381,0.0004829137,0.0001542925],"about_ca_system_candidate":false,"about_ca_system_consensus":false,"about_ca_system_score_codex":0.0002858533,"about_ca_system_score_gemma":0.001572331,"about_ca_topic_candidate":false,"about_ca_topic_consensus":false,"about_ca_topic_score_codex":0.00009365633,"about_ca_topic_score_gemma":0.0001091298,"domain_scores_codex":[0.9950728,0.0003518787,0.0008985481,0.0007066161,0.002195303,0.0007748496],"domain_scores_gemma":[0.9948661,0.001935172,0.0001866407,0.0007440276,0.001897824,0.0003702489],"domain_codex":null,"domain_gemma":null,"domain_candidate":null,"domain_consensus":null,"study_design_codex":"observational","study_design_gemma":"observational","study_design_scores_codex":[0.08769882,0.00855171,0.6722481,0.003295662,0.0008963725,0.00001354183,0.0010146,0.00006413671,0.01175974,0.001878891,0.01003748,0.2025409],"study_design_scores_gemma":[0.0412335,0.01386509,0.8964677,0.001767346,0.00005803581,0.000003102033,0.0004460377,0.002224517,0.01237997,0.000658385,0.03045014,0.0004461529],"study_design_candidate":"observational","study_design_consensus":"observational","genre_codex":"empirical","genre_gemma":"empirical","genre_scores_codex":[0.9877895,0.00007866303,0.00135176,0.0003339143,0.0001280258,0.008828568,0.00005262935,0.00002421487,0.001412785],"genre_scores_gemma":[0.8636476,0.00001549623,0.1283961,0.00002851654,0.0000516479,0.000558966,0.0002423923,0.00005789665,0.007001365],"genre_candidate":"empirical","genre_consensus":"empirical","teacher_disagreement_score":0.2242196,"threshold_uncertainty_score":0.612537,"prediction_status":"machine_predicted_unvalidated"},"machine_scores":{"provisional":true,"baseline":true,"maturity_gate_passed":false,"score_opus":0.126589237287119,"score_gpt":0.4641736846248831,"score_spread":0.3375844473377642,"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."}}