{"id":"W4411636199","doi":"10.1208/s12248-025-01093-y","title":"Pharmacokinetics and Pharmacodynamics of Nomlabofusp in Non-clinical Studies of Friedreich’s Ataxia","year":2025,"lang":"en","type":"article","venue":"The AAPS Journal","topic":"Genetic Neurodegenerative Diseases","field":"Neuroscience","cited_by":2,"is_retracted":false,"has_abstract":true,"ca_institutions":"Altasciences (Canada)","funders":"National Center for Advancing Translational Sciences; National Institutes of Health; Frederick National Laboratory for Cancer Research","keywords":"Frataxin; Ataxia; Pharmacokinetics; Pharmacodynamics; Pharmacology; Knockout mouse; Medicine; Biology; Endocrinology; Internal medicine; Neuroscience; Mitochondrion; Cell biology; Receptor","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":[0.0005810317,0.0001206382,0.0003034628,0.000124251,0.00008715573,0.00001611415,0.00037308,0.00002333907,0.00001873139],"category_scores_gemma":[0.0009748958,0.00008302797,0.00007474874,0.0003322237,0.0005743411,0.0000549369,0.000215857,0.0003295627,0.0000013739],"about_ca_system_candidate":false,"about_ca_system_consensus":false,"about_ca_system_score_codex":0.00001352863,"about_ca_system_score_gemma":0.00009764261,"about_ca_topic_candidate":false,"about_ca_topic_consensus":false,"about_ca_topic_score_codex":0.000002312121,"about_ca_topic_score_gemma":0.000003434251,"domain_scores_codex":[0.9983606,0.0004380853,0.0006264697,0.0001851779,0.000219915,0.0001697039],"domain_scores_gemma":[0.9985899,0.0007530529,0.0002806415,0.0001810866,0.0001372318,0.00005806619],"domain_codex":null,"domain_gemma":null,"domain_candidate":null,"domain_consensus":null,"study_design_codex":"bench_or_experimental","study_design_gemma":"bench_or_experimental","study_design_scores_codex":[0.0002070145,0.0002488458,0.02927148,0.00007671264,0.00005856706,0.00002516743,0.0004937936,0.001057566,0.9624377,0.0003695002,0.001461085,0.004292504],"study_design_scores_gemma":[0.002119796,0.0001485184,0.05044387,0.0001122663,0.00013821,0.00003377799,0.0003064419,0.003646623,0.9404345,0.002179388,0.0003240151,0.0001126506],"study_design_candidate":"bench_or_experimental","study_design_consensus":"bench_or_experimental","genre_codex":"empirical","genre_gemma":"empirical","genre_scores_codex":[0.9966499,0.001403969,0.0001697236,0.0007408354,0.0005462529,0.0001519055,0.00002249605,0.000003679313,0.0003111933],"genre_scores_gemma":[0.9929913,0.005953833,0.0001644974,0.000659303,0.00006449074,0.00000288457,1.581903e-7,0.000008212979,0.0001553405],"genre_candidate":"empirical","genre_consensus":"empirical","teacher_disagreement_score":0.02200332,"threshold_uncertainty_score":0.3385783,"prediction_status":"machine_predicted_unvalidated"},"machine_scores":{"provisional":true,"baseline":true,"maturity_gate_passed":false,"score_opus":0.07450655621278808,"score_gpt":0.4234999634098038,"score_spread":0.3489934071970158,"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."}}