{"id":"W3203748062","doi":"10.1017/cjn.2019.166","title":"P.066 AVXS-101 gene-replacement therapy (GRT) in spinal muscular atrophy type 1 (SMA1): long-term follow-up from the phase 1 clinical trial","year":2019,"lang":"en","type":"article","venue":"Canadian Journal of Neurological Sciences / Journal Canadien des Sciences Neurologiques","topic":"Neurogenetic and Muscular Disorders Research","field":"Medicine","cited_by":2,"is_retracted":false,"has_abstract":true,"ca_institutions":"","funders":"","keywords":"Medicine; Cohort; Adverse effect; Spinal muscular atrophy; Natural history study; Clinical trial; Pediatrics; Cohort study; Natural history; Internal medicine; Disease; Physical therapy; Surgery","routes":{"ca_aff":false,"ca_fund":false,"ca_venue":true,"about_ca":false,"invisible_to_affiliation_only":true},"retraction":null,"screen":null,"direct_labels":[],"prediction":{"model_version":"metacan-v3-hybrid-931329e0061c","candidate_categories":[],"consensus_categories":[],"category_scores_codex":[0.0019388,0.0005249598,0.0007325107,0.0002184923,0.0004931189,0.001213821,0.0004822978,0.001611284,0.01825112],"category_scores_gemma":[0.00186591,0.0001813702,0.00105924,0.0004403932,0.0004114686,0.0007311479,0.0002538421,0.00251795,0.003065939],"about_ca_system_candidate":false,"about_ca_system_consensus":false,"about_ca_system_score_codex":0.000398323,"about_ca_system_score_gemma":0.0009327608,"about_ca_topic_candidate":false,"about_ca_topic_consensus":false,"about_ca_topic_score_codex":0.000277336,"about_ca_topic_score_gemma":0.0008509835,"domain_scores_codex":[0.9995278,0.0002002019,0.00003056345,0.00007259358,0.0001044691,0.00006429477],"domain_scores_gemma":[0.9988048,0.0003502685,0.0002597808,0.00007518521,0.0001536383,0.0003563066],"domain_codex":null,"domain_gemma":null,"domain_candidate":null,"domain_consensus":null,"study_design_codex":"randomized_trial","study_design_gemma":"nonrandomized_trial","study_design_scores_codex":[0.4751111,0.01470921,0.009697099,0.002957445,0.002670645,0.000649672,0.0002058942,0.001701351,0.01498873,0.001435492,0.119611,0.3562624],"study_design_scores_gemma":[0.6435223,0.2325435,0.04513306,0.0005629167,0.002012841,0.001149842,0.0001022306,0.001985257,0.005506577,0.00240349,0.06501855,0.00005949076],"study_design_candidate":"nonrandomized_trial","study_design_consensus":null,"genre_codex":"empirical","genre_gemma":"empirical","genre_scores_codex":[0.8536337,0.02424216,0.002972905,0.01786254,0.002654052,0.005737048,0.02776199,0.0008579714,0.06427773],"genre_scores_gemma":[0.9363542,0.007961278,0.002927927,0.009593592,0.001475981,0.005001242,0.01342189,0.0001335929,0.02313038],"genre_candidate":"empirical","genre_consensus":"empirical","teacher_disagreement_score":0.01825112,"threshold_uncertainty_score":0.06105608,"prediction_status":"machine_predicted_unvalidated"},"machine_scores":{"provisional":true,"baseline":true,"maturity_gate_passed":false,"score_opus":0.1019666225384174,"score_gpt":0.3876843733114433,"score_spread":0.2857177507730259,"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."}}