{"id":"W4210398641","doi":"10.1016/j.ymgme.2021.11.171","title":"Mini-COMET study: Safety, biomarker, and efficacy data after avalglucosidase alfa dosing for ≥ 97 weeks in participants with infantile-onset pompe disease (IOPD) previously treated with alglucosidase alfa who had demonstrated clinical decline","year":2022,"lang":"en","type":"article","venue":"Molecular Genetics and Metabolism","topic":"Lysosomal Storage Disorders Research","field":"Medicine","cited_by":1,"is_retracted":false,"has_abstract":false,"ca_institutions":"Sanofi (Canada)","funders":"","keywords":"Dosing; Medicine; Internal medicine; Pediatrics; Oncology","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":[0.003311768,0.001004357,0.002337701,0.0004876388,0.0005739672,0.00123869,0.0005339126,0.001376133,0.003666787],"category_scores_gemma":[0.003629306,0.0004529933,0.002833469,0.0004416186,0.0004848935,0.00127181,0.0004384249,0.002065186,0.0008993076],"about_ca_system_candidate":false,"about_ca_system_consensus":false,"about_ca_system_score_codex":0.0005191554,"about_ca_system_score_gemma":0.0004001855,"about_ca_topic_candidate":false,"about_ca_topic_consensus":false,"about_ca_topic_score_codex":0.0005034061,"about_ca_topic_score_gemma":0.0008206907,"domain_scores_codex":[0.9991612,0.0003459134,0.00007917194,0.00018289,0.0001457775,0.00008499484],"domain_scores_gemma":[0.9967762,0.0007731444,0.001167765,0.0003263313,0.0003574777,0.0005991171],"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":[0.8462693,0.004480836,0.07750083,0.001008822,0.007752054,0.0002031262,0.0002406639,0.0004778834,0.01021694,0.0001613673,0.001954442,0.04973364],"study_design_scores_gemma":[0.255251,0.2146368,0.4985781,0.0003786353,0.01288333,0.001422049,0.0002823931,0.001911487,0.007060821,0.0008254439,0.006590117,0.0001796358],"study_design_candidate":"nonrandomized_trial","study_design_consensus":null,"genre_codex":"empirical","genre_gemma":"empirical","genre_scores_codex":[0.9904031,0.003468481,0.0005285462,0.0002980775,0.0001047479,0.0005067157,0.002984484,0.0001083866,0.001597561],"genre_scores_gemma":[0.9920135,0.0007271349,0.0007350118,0.0005876115,0.0001423264,0.0007107526,0.003422531,0.00002568893,0.001635332],"genre_candidate":"empirical","genre_consensus":"empirical","teacher_disagreement_score":0.003666787,"threshold_uncertainty_score":0.01751453,"prediction_status":"machine_predicted_unvalidated"},"machine_scores":{"provisional":true,"baseline":true,"maturity_gate_passed":false,"score_opus":0.0524031344927647,"score_gpt":0.3537848493898312,"score_spread":0.3013817148970666,"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."}}