{"id":"W4405897742","doi":"10.1212/wnl.0000000000210268","title":"Safety and Efficacy of IV Onasemnogene Abeparvovec for Pediatric Patients With Spinal Muscular Atrophy","year":2024,"lang":"en","type":"article","venue":"Neurology","topic":"Neurogenetic and Muscular Disorders Research","field":"Medicine","cited_by":23,"is_retracted":false,"has_abstract":true,"ca_institutions":"Children's Hospital of Eastern Ontario; University of Ottawa","funders":"Chugai Pharmaceutical; Institut National de la Santé et de la Recherche Médicale; Sarepta Therapeutics; European Commission; Sanofi; PTC Therapeutics; AveXis; Biogen; Amicus Therapeutics; Regeneron Pharmaceuticals; Alexion Pharmaceuticals; Pfizer","keywords":"Spinal muscular atrophy; Medicine; Atrophy; Progressive muscular atrophy; Physical medicine and rehabilitation; Pediatrics; Physical therapy; Internal medicine; Amyotrophic lateral sclerosis; Disease","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":[0.000077619,0.0001191123,0.0002441659,0.0001316812,0.00005788089,0.00001024017,0.00007719552,0.00007582824,0.00002748374],"category_scores_gemma":[0.00007756179,0.00009069902,0.00009189743,0.0001311914,0.0001380938,0.00002067028,0.00006394627,0.0001607136,0.000006151993],"about_ca_system_candidate":false,"about_ca_system_consensus":false,"about_ca_system_score_codex":0.000004825147,"about_ca_system_score_gemma":0.0000855679,"about_ca_topic_candidate":false,"about_ca_topic_consensus":false,"about_ca_topic_score_codex":0.00001176402,"about_ca_topic_score_gemma":0.000004236806,"domain_scores_codex":[0.9989702,0.00003880143,0.0001793614,0.0003573647,0.0002116189,0.0002426589],"domain_scores_gemma":[0.9994174,0.00008821821,0.00003532422,0.0002669569,0.00008574269,0.0001063259],"domain_codex":null,"domain_gemma":null,"domain_candidate":null,"domain_consensus":null,"study_design_codex":"observational","study_design_gemma":"observational","study_design_scores_codex":[0.04723569,0.001495055,0.8264411,0.00336435,0.0006584932,0.0005213976,0.0001548561,0.00007081257,0.005168891,0.001776326,0.0007585418,0.1123545],"study_design_scores_gemma":[0.005875398,0.01098732,0.9694021,0.00001317336,0.0003139342,0.00003009232,0.00000142019,0.0002920707,0.0001892299,0.00004175413,0.01275628,0.00009720461],"study_design_candidate":"observational","study_design_consensus":"observational","genre_codex":"empirical","genre_gemma":"empirical","genre_scores_codex":[0.9958609,0.001558263,0.000514848,0.001006267,0.0001108958,0.0007695882,0.0000203,0.00002208812,0.000136823],"genre_scores_gemma":[0.9988842,0.0003671205,0.0002188791,0.000306089,0.0001137717,0.00001998357,0.00001954337,0.00003377285,0.00003665493],"genre_candidate":"empirical","genre_consensus":"empirical","teacher_disagreement_score":0.142961,"threshold_uncertainty_score":0.3698598,"prediction_status":"machine_predicted_unvalidated"},"machine_scores":{"provisional":true,"baseline":true,"maturity_gate_passed":false,"score_opus":0.009055922467832234,"score_gpt":0.2718110679125739,"score_spread":0.2627551454447417,"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."}}