{"id":"W4286008638","doi":"10.1093/brain/awac252","title":"Effect of nusinersen on motor, respiratory and bulbar function in early-onset spinal muscular atrophy","year":2022,"lang":"en","type":"article","venue":"Brain","topic":"Neurogenetic and Muscular Disorders Research","field":"Medicine","cited_by":70,"is_retracted":false,"has_abstract":true,"ca_institutions":"Ottawa Hospital; Children's Hospital of Eastern Ontario; University of Ottawa","funders":"Eisai; Albert-Ludwigs-Universität Freiburg; Canada Research Chairs; Muscular Dystrophy Canada; Sarepta Therapeutics; Canadian Institutes of Health Research; GW Pharmaceuticals; Sanofi; PTC Therapeutics; Ultragenyx Pharmaceutical; AveXis; Biogen; Alnylam Pharmaceuticals; F. Hoffmann-La Roche; Alexion Pharmaceuticals; Pfizer","keywords":"Spinal muscular atrophy; Medicine; Pediatrics; Atrophy; Weakness; Progressive muscular atrophy; Neuromuscular disease; Clinical trial; Physical therapy; Physical medicine and rehabilitation; Surgery; Disease; Internal medicine; Amyotrophic lateral sclerosis","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":[],"consensus_categories":[],"category_scores_codex":[0.0006217079,0.00009445209,0.0001941798,0.000244499,0.00005673903,0.000004485421,0.0000513776,0.00003442829,0.00008857381],"category_scores_gemma":[0.0001418448,0.00008580918,0.00007753948,0.0002471152,0.00006623385,0.00001469826,0.00007543544,0.0002414307,0.000007553006],"about_ca_system_candidate":false,"about_ca_system_consensus":false,"about_ca_system_score_codex":0.00003625424,"about_ca_system_score_gemma":0.0000549677,"about_ca_topic_candidate":false,"about_ca_topic_consensus":false,"about_ca_topic_score_codex":0.00007438166,"about_ca_topic_score_gemma":0.000003459017,"domain_scores_codex":[0.9986745,0.0003991241,0.000139085,0.0002307146,0.0003870759,0.0001695257],"domain_scores_gemma":[0.999582,0.00007462782,0.00003016373,0.0002256194,0.00001732285,0.00007028707],"domain_codex":null,"domain_gemma":null,"domain_candidate":null,"domain_consensus":null,"study_design_codex":"observational","study_design_gemma":"observational","study_design_scores_codex":[0.0302676,0.0009831859,0.3936083,0.001408485,0.000259984,0.001180422,0.000565496,0.0001855709,0.3627502,0.0003278777,0.007996134,0.2004668],"study_design_scores_gemma":[0.007993801,0.03268478,0.9080074,0.0000796008,0.00006919154,0.00005163243,0.0001274088,0.0001572279,0.004670879,0.0000476172,0.04593316,0.0001772288],"study_design_candidate":"observational","study_design_consensus":"observational","genre_codex":"empirical","genre_gemma":"empirical","genre_scores_codex":[0.9977428,0.0008068125,0.00001538253,0.0003852548,0.00005428724,0.0006056718,0.000006248912,0.00001263507,0.0003709211],"genre_scores_gemma":[0.998926,0.00001149863,0.00001202544,0.0007181008,0.00003171159,0.00007475946,0.00000635254,0.00001904672,0.0002005272],"genre_candidate":"empirical","genre_consensus":"empirical","teacher_disagreement_score":0.5143992,"threshold_uncertainty_score":0.3499197,"prediction_status":"machine_predicted_unvalidated"},"machine_scores":{"provisional":true,"baseline":true,"maturity_gate_passed":false,"score_opus":0.01255415004248971,"score_gpt":0.2901318235974736,"score_spread":0.2775776735549839,"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."}}