{"id":"W4307106827","doi":"10.1186/s13023-022-02547-8","title":"Improved upper limb function in non-ambulant children with SMA type 2 and 3 during nusinersen treatment: a prospective 3-years SMArtCARE registry study","year":2022,"lang":"en","type":"article","venue":"Orphanet Journal of Rare Diseases","topic":"Neurogenetic and Muscular Disorders Research","field":"Medicine","cited_by":40,"is_retracted":false,"has_abstract":true,"ca_institutions":"Ottawa Hospital; Children's Hospital of Eastern Ontario; University of Ottawa","funders":"Albert-Ludwigs-Universität Freiburg; Harmony Biosciences; Sarepta Therapeutics; F. Hoffmann-La Roche; Argenx; Biogen; Spark Therapeutics; PTC Therapeutics; Ultragenyx Pharmaceutical; Sanofi; Pfizer","keywords":"SMA*; Medicine; Spinal muscular atrophy; Milestone; Motor function; Pediatrics; Physical therapy; Disease; Neuromuscular disease; Physical medicine and rehabilitation; Internal medicine","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":"metacan-v3-hybrid-931329e0061c","candidate_categories":[],"consensus_categories":[],"category_scores_codex":[0.001555106,0.0005122909,0.0009522869,0.001304068,0.0006328108,0.00108097,0.0006451737,0.0007320825,0.001455527],"category_scores_gemma":[0.00273204,0.0004331083,0.001382798,0.002230765,0.0003541847,0.0009456208,0.001019581,0.0007268566,0.0004673982],"about_ca_system_candidate":false,"about_ca_system_consensus":false,"about_ca_system_score_codex":0.0008589841,"about_ca_system_score_gemma":0.0008921139,"about_ca_topic_candidate":false,"about_ca_topic_consensus":false,"about_ca_topic_score_codex":0.006792344,"about_ca_topic_score_gemma":0.009896695,"domain_scores_codex":[0.9985508,0.0003094524,0.00023431,0.0004114199,0.0002738069,0.0002202592],"domain_scores_gemma":[0.9962561,0.0003726429,0.002250031,0.0003484734,0.0003753786,0.0003972335],"domain_codex":null,"domain_gemma":null,"domain_candidate":null,"domain_consensus":null,"study_design_codex":"observational","study_design_gemma":"observational","study_design_scores_codex":[0.000391202,0.00006058163,0.998173,0.00002127128,0.0001325352,0.0001325574,0.00008733718,0.00003041455,0.00007443798,0.000009771926,0.000143653,0.0007432647],"study_design_scores_gemma":[0.0000415208,0.0002662967,0.9987613,0.00001243275,0.00007820697,0.0003577523,0.0001479103,0.00005959645,0.00003664438,0.000007240371,0.0002250007,0.00000617052],"study_design_candidate":"observational","study_design_consensus":"observational","genre_codex":"empirical","genre_gemma":"empirical","genre_scores_codex":[0.9968597,0.0002551899,0.00004651021,0.00002005892,0.000004106181,0.00003423732,0.002511707,0.000003771024,0.0002648123],"genre_scores_gemma":[0.9920924,0.0002505627,0.0001644744,0.00004984748,0.00001462811,0.0001082035,0.007098513,0.000006536048,0.0002148192],"genre_candidate":"empirical","genre_consensus":"empirical","teacher_disagreement_score":0.006792344,"threshold_uncertainty_score":0.01350558,"prediction_status":"machine_predicted_unvalidated"},"machine_scores":{"provisional":true,"baseline":true,"maturity_gate_passed":false,"score_opus":0.007109677072452832,"score_gpt":0.2477435460918635,"score_spread":0.2406338690194106,"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."}}