{"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":"metacan-v3-hybrid-931329e0061c","candidate_categories":[],"consensus_categories":[],"category_scores_codex":[0.003825165,0.001633921,0.00178946,0.0009196251,0.001229433,0.002773837,0.001681406,0.008613528,0.005394263],"category_scores_gemma":[0.007824846,0.0005055934,0.001295148,0.0005920841,0.0009837192,0.001346395,0.0005182432,0.01175537,0.00326188],"about_ca_system_candidate":false,"about_ca_system_consensus":false,"about_ca_system_score_codex":0.001305964,"about_ca_system_score_gemma":0.001315038,"about_ca_topic_candidate":false,"about_ca_topic_consensus":false,"about_ca_topic_score_codex":0.0008177529,"about_ca_topic_score_gemma":0.002393455,"domain_scores_codex":[0.998794,0.00025331,0.0001941189,0.0001518503,0.0004947881,0.000111987],"domain_scores_gemma":[0.994538,0.003108953,0.0002337573,0.000113832,0.001319424,0.0006859307],"domain_codex":null,"domain_gemma":null,"domain_candidate":null,"domain_consensus":null,"study_design_codex":"not_applicable","study_design_gemma":"case_report","study_design_scores_codex":[0.0002784533,0.00002843893,0.00003681647,0.0003791725,0.00004191226,0.0004679564,0.00001351794,0.00004667751,0.0003245909,0.0004827656,0.9797506,0.01814911],"study_design_scores_gemma":[0.0004859885,0.0002287887,0.00100571,0.0005349431,0.0001727387,0.001093801,0.00003640674,0.0003316214,0.0006816201,0.001270157,0.9941274,0.00003088486],"study_design_candidate":"case_report","study_design_consensus":null,"genre_codex":"editorial","genre_gemma":"editorial","genre_scores_codex":[0.0004140064,0.01825016,0.000329156,0.05450938,0.9220218,0.00003716745,0.0001102456,0.00009273709,0.004235388],"genre_scores_gemma":[0.003204828,0.01415056,0.0002417322,0.04261832,0.9240744,0.00004757316,0.00006981627,0.00006478698,0.01552801],"genre_candidate":"editorial","genre_consensus":"editorial","teacher_disagreement_score":0.008613528,"threshold_uncertainty_score":0.02022964,"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."}}