{"id":"W4404584233","doi":"10.1186/s13023-024-03421-5","title":"Genotype-specific effects of elamipretide in patients with primary mitochondrial myopathy: a post hoc analysis of the MMPOWER-3 trial","year":2024,"lang":"en","type":"article","venue":"Orphanet Journal of Rare Diseases","topic":"Mitochondrial Function and Pathology","field":"Biochemistry, Genetics and Molecular Biology","cited_by":10,"is_retracted":false,"has_abstract":true,"ca_institutions":"McMaster University; McMaster Children's Hospital; Vancouver General Hospital","funders":"National Center for Advancing Translational Sciences","keywords":"Mitochondrial DNA; Post-hoc analysis; Cohort; Mitochondrial myopathy; Population; Biology; Genetics; Chronic progressive external ophthalmoplegia; Mitochondrial disease; Internal medicine; Genotype; Placebo; Medicine; Pathology; Gene","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":"metacan-v3-hybrid-931329e0061c","candidate_categories":[],"consensus_categories":[],"category_scores_codex":[0.004057792,0.0009839756,0.00266466,0.0002074336,0.0002544439,0.0009488567,0.0004933477,0.0008821646,0.003144121],"category_scores_gemma":[0.004609424,0.0002206912,0.003326517,0.0002669333,0.0004773399,0.0007980668,0.0004601477,0.001691588,0.0003641571],"about_ca_system_candidate":false,"about_ca_system_consensus":false,"about_ca_system_score_codex":0.0003722943,"about_ca_system_score_gemma":0.0005474138,"about_ca_topic_candidate":false,"about_ca_topic_consensus":false,"about_ca_topic_score_codex":0.0003128275,"about_ca_topic_score_gemma":0.00070385,"domain_scores_codex":[0.9977247,0.00147443,0.0001603334,0.0002606681,0.0001107889,0.0002691335],"domain_scores_gemma":[0.9959708,0.002220611,0.000725803,0.0004896573,0.0002126708,0.0003804838],"domain_codex":null,"domain_gemma":null,"domain_candidate":null,"domain_consensus":null,"study_design_codex":"randomized_trial","study_design_gemma":"observational","study_design_scores_codex":[0.9782419,0.0008685151,0.005447524,0.0002473355,0.004169375,0.00008049083,0.00005800563,0.0004406462,0.002985433,0.00007382684,0.0003937692,0.006993158],"study_design_scores_gemma":[0.4565307,0.3727048,0.1429763,0.0001315342,0.01572321,0.0002681583,0.0002220378,0.003573868,0.004106036,0.0009006982,0.002740246,0.0001223969],"study_design_candidate":"observational","study_design_consensus":null,"genre_codex":"empirical","genre_gemma":"empirical","genre_scores_codex":[0.9961325,0.0016511,0.000326799,0.0001543627,0.0001009628,0.0003848191,0.0004738076,0.00002578492,0.0007498699],"genre_scores_gemma":[0.9973781,0.0002985226,0.0003986004,0.0002049874,0.00009773223,0.0004858864,0.0004413966,0.00001531493,0.0006795371],"genre_candidate":"empirical","genre_consensus":"empirical","teacher_disagreement_score":0.004057792,"threshold_uncertainty_score":0.02145994,"prediction_status":"machine_predicted_unvalidated"},"machine_scores":{"provisional":true,"baseline":true,"maturity_gate_passed":false,"score_opus":0.00344311847009756,"score_gpt":0.2063202761584206,"score_spread":0.202877157688323,"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."}}