{"id":"W4388730923","doi":"10.1002/mus.28000","title":"Sustained clinical benefit following systemic gene replacement therapy in <scp>Duchenne</scp> muscular dystrophy","year":2023,"lang":"en","type":"editorial","venue":"Muscle & Nerve","topic":"Muscle Physiology and Disorders","field":"Biochemistry, Genetics and Molecular Biology","cited_by":1,"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; Muscular Dystrophy Canada; Canada Research Chairs; Argenx; Harmony Biosciences; Spark Therapeutics; Sarepta Therapeutics; F. Hoffmann-La Roche; Biogen; Sanofi; PTC Therapeutics; Ultragenyx Pharmaceutical; Pfizer","keywords":"Duchenne muscular dystrophy; Medicine; Genetic enhancement; Muscular dystrophy; Gene; Internal medicine; Biology; Genetics","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":["metaepi_narrow","research_integrity"],"consensus_categories":[],"category_scores_codex":[0.001527555,0.0007162789,0.001028913,0.0001914525,0.0001664879,0.0000556427,0.0007601294,0.002327297,0.00001072994],"category_scores_gemma":[0.002311264,0.0007150245,0.001038821,0.0003222368,0.0001071221,0.000009415424,0.0005662197,0.0009664708,0.00005433668],"about_ca_system_candidate":false,"about_ca_system_consensus":false,"about_ca_system_score_codex":0.0001080459,"about_ca_system_score_gemma":0.0004721089,"about_ca_topic_candidate":false,"about_ca_topic_consensus":false,"about_ca_topic_score_codex":0.0002685311,"about_ca_topic_score_gemma":0.0001503685,"domain_scores_codex":[0.9950742,0.0004878808,0.001190041,0.001687528,0.000555435,0.001004977],"domain_scores_gemma":[0.997029,0.0006335104,0.0004567372,0.001434152,0.000225904,0.0002206738],"domain_codex":null,"domain_gemma":null,"domain_candidate":null,"domain_consensus":null,"study_design_codex":"not_applicable","study_design_gemma":"not_applicable","study_design_scores_codex":[0.0005379464,0.0004291514,0.0007552968,0.0002877047,0.001007828,0.0002154656,0.0002045818,0.0004212083,0.05432934,0.00003033836,0.9383685,0.003412665],"study_design_scores_gemma":[0.01171052,0.003363594,0.003330101,0.0003643491,0.0002162997,0.000003345373,0.001542645,0.00008461719,0.002435782,0.0003691921,0.9756559,0.0009236987],"study_design_candidate":"not_applicable","study_design_consensus":"not_applicable","genre_codex":"empirical","genre_gemma":"editorial","genre_scores_codex":[0.6454567,0.003418009,0.00005830232,0.00003343182,0.3499697,0.0006738185,0.0001173753,0.00007521669,0.000197365],"genre_scores_gemma":[0.4562684,0.007133007,0.0001754183,0.0002103199,0.5234909,0.0007010943,0.008559546,0.0004154248,0.003045881],"genre_candidate":"empirical","genre_consensus":null,"teacher_disagreement_score":0.1891883,"threshold_uncertainty_score":0.9995301,"prediction_status":"machine_predicted_unvalidated"},"machine_scores":{"provisional":true,"baseline":true,"maturity_gate_passed":false,"score_opus":0.01370649357821319,"score_gpt":0.2896962788418377,"score_spread":0.2759897852636246,"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."}}