{"id":"W4403070265","doi":"10.20944/preprints202409.2440.v1","title":"Recent Progress of Antisense Oligonucleotide Therapy for Superoxide Dismutase 1-mutated Amyotrophic Lateral Sclerosis: Focus on Tofersen","year":2024,"lang":"en","type":"preprint","venue":"Preprints.org","topic":"Amyotrophic Lateral Sclerosis Research","field":"Medicine","cited_by":6,"is_retracted":false,"has_abstract":true,"ca_institutions":"","funders":"Faculty of Medicine and Dentistry, University of Alberta; Canadian Institutes of Health Research; Muscular Dystrophy Canada; University of Alberta; Alberta Innovates - Health Solutions; Women and Children's Health Research Institute; Children's Health Research Institute; Alberta Innovates; Heart and Stroke Foundation of Canada; U.S. Department of Defense","keywords":"Amyotrophic lateral sclerosis; Superoxide dismutase; Oligonucleotide; Focus (optics); Medicine; Cancer research; Neuroscience; Biology; Genetics; Pathology; Disease; Internal medicine; Gene; Oxidative stress; Physics","routes":{"ca_aff":false,"ca_fund":true,"ca_venue":false,"about_ca":false,"invisible_to_affiliation_only":true},"retraction":null,"screen":null,"direct_labels":[],"prediction":{"model_version":"codex-gemma-dda1882f352a","candidate_categories":["metaepi_narrow","insufficient_payload"],"consensus_categories":[],"category_scores_codex":[0.001092786,0.001012771,0.001724009,0.0006318801,0.000141499,0.00008520443,0.000815678,0.0008163314,0.0008613312],"category_scores_gemma":[0.0003771566,0.0008307617,0.0009053705,0.0005396785,0.0005829449,0.00009289919,0.002198287,0.002272221,0.0009417895],"about_ca_system_candidate":false,"about_ca_system_consensus":false,"about_ca_system_score_codex":0.0006332051,"about_ca_system_score_gemma":0.000656988,"about_ca_topic_candidate":false,"about_ca_topic_consensus":false,"about_ca_topic_score_codex":0.0002903478,"about_ca_topic_score_gemma":0.00001363891,"domain_scores_codex":[0.9932552,0.0003850876,0.001498324,0.002255429,0.001353187,0.001252751],"domain_scores_gemma":[0.9954799,0.0001940325,0.0004354008,0.002461721,0.0008070926,0.0006218743],"domain_codex":null,"domain_gemma":null,"domain_candidate":null,"domain_consensus":null,"study_design_codex":"bench_or_experimental","study_design_gemma":"observational","study_design_scores_codex":[0.01872849,0.004036592,0.2156211,0.004491761,0.004577704,0.0008755601,0.002944878,0.0005327606,0.6852685,0.0006976803,0.001313532,0.06091152],"study_design_scores_gemma":[0.005601723,0.001202666,0.60356,0.005797321,0.0004743764,0.00004732946,0.0000543611,0.0008168634,0.3746835,0.004830468,0.001899097,0.001032337],"study_design_candidate":"bench_or_experimental","study_design_consensus":null,"genre_codex":"empirical","genre_gemma":"empirical","genre_scores_codex":[0.9792367,0.002063853,0.000005879112,0.009985281,0.0008467938,0.00627112,0.0004563542,0.0004907482,0.0006432573],"genre_scores_gemma":[0.9864991,0.009464065,0.0003444129,0.0002934909,0.0005057184,0.001364143,0.0003736995,0.000333548,0.0008218663],"genre_candidate":"empirical","genre_consensus":"empirical","teacher_disagreement_score":0.3879389,"threshold_uncertainty_score":0.9998361,"prediction_status":"machine_predicted_unvalidated"},"machine_scores":{"provisional":true,"baseline":true,"maturity_gate_passed":false,"score_opus":0.1739005149002207,"score_gpt":0.3867381783198471,"score_spread":0.2128376634196265,"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."}}