{"id":"W3035514338","doi":"10.3233/jnd-200534","title":"Measuring Outcomes in Adults with Spinal Muscular Atrophy – Challenges and Future Directions – Meeting Report","year":2020,"lang":"en","type":"article","venue":"Journal of Neuromuscular Diseases","topic":"Neurogenetic and Muscular Disorders Research","field":"Medicine","cited_by":67,"is_retracted":false,"has_abstract":true,"ca_institutions":"Ottawa Hospital; Children's Hospital of Eastern Ontario; University of Ottawa","funders":"Audentes Therapeutics; Sarepta Therapeutics; GW Pharmaceuticals; Ionis Pharmaceuticals; Sanofi; PTC Therapeutics; Ultragenyx Pharmaceutical; CSL Behring; AveXis; Biogen; Alnylam Pharmaceuticals; Alexion Pharmaceuticals; Pfizer","keywords":"SMN1; Spinal muscular atrophy; SMA*; Medicine; Motor neuron; Neuromuscular disease; Clinical trial; Population; Disease; Quality of life (healthcare); Physical medicine and rehabilitation; Bioinformatics; Physical therapy; Internal medicine; Biology","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":[0.03289133,0.0009577405,0.001555568,0.00156797,0.0008132732,0.00381674,0.00224971,0.00241251,0.010304],"category_scores_gemma":[0.07726637,0.0002833353,0.001459611,0.001900429,0.0006980534,0.004700154,0.003654839,0.004300948,0.003984071],"about_ca_system_candidate":false,"about_ca_system_consensus":false,"about_ca_system_score_codex":0.002189784,"about_ca_system_score_gemma":0.006103121,"about_ca_topic_candidate":false,"about_ca_topic_consensus":false,"about_ca_topic_score_codex":0.006306393,"about_ca_topic_score_gemma":0.007997647,"domain_scores_codex":[0.9884464,0.006230121,0.001560558,0.0008206296,0.002247499,0.0006948505],"domain_scores_gemma":[0.9370139,0.0173234,0.005198999,0.001743697,0.02919307,0.009526813],"domain_codex":null,"domain_gemma":null,"domain_candidate":null,"domain_consensus":null,"study_design_codex":"design_other","study_design_gemma":"not_applicable","study_design_scores_codex":[0.0002725254,0.0001637307,0.02796633,0.003623701,0.000251198,0.0001997143,0.000617214,0.0001985242,0.0001489097,0.001884123,0.426425,0.5382491],"study_design_scores_gemma":[0.0002878123,0.0009666709,0.1633184,0.04307109,0.0008666351,0.001697581,0.00623249,0.001298208,0.0003890725,0.01759916,0.7639617,0.0003111805],"study_design_candidate":"not_applicable","study_design_consensus":null,"genre_codex":"commentary","genre_gemma":"commentary","genre_scores_codex":[0.01202513,0.3459044,0.004909021,0.5721691,0.03845596,0.0003967214,0.00476055,0.0006083795,0.02077086],"genre_scores_gemma":[0.1874181,0.5734242,0.03527698,0.0962646,0.07944465,0.002306598,0.01561207,0.0004749376,0.009777952],"genre_candidate":"commentary","genre_consensus":"commentary","teacher_disagreement_score":0.03289133,"threshold_uncertainty_score":0.173948,"prediction_status":"machine_predicted_unvalidated"},"machine_scores":{"provisional":true,"baseline":true,"maturity_gate_passed":false,"score_opus":0.03291156471814895,"score_gpt":0.2714065526890334,"score_spread":0.2384949879708844,"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."}}