{"id":"W4400901227","doi":"10.1016/s1474-4422(24)00216-3","title":"Safety, tolerability, and pharmacokinetics of antisense oligonucleotide BIIB078 in adults with C9orf72-associated amyotrophic lateral sclerosis: a phase 1, randomised, double blinded, placebo-controlled, multiple ascending dose study","year":2024,"lang":"en","type":"article","venue":"The Lancet Neurology","topic":"Amyotrophic Lateral Sclerosis Research","field":"Medicine","cited_by":70,"is_retracted":false,"has_abstract":false,"ca_institutions":"Health Sciences Centre; University of Toronto; Sunnybrook Health Science Centre; University of Calgary; Montreal Neurological Institute and Hospital","funders":"Biogen","keywords":"Amyotrophic lateral sclerosis; Tolerability; Pharmacokinetics; Placebo; Medicine; C9orf72; Internal medicine; Pharmacology; Adverse effect; Pathology; Disease","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":[0.0009543648,0.001850998,0.002542471,0.0003315203,0.0003708141,0.0007730088,0.0005717787,0.00189859,0.002734729],"category_scores_gemma":[0.001121815,0.0007496632,0.001072013,0.0002926571,0.0008103267,0.001102272,0.0002421887,0.002532025,0.0008012334],"about_ca_system_candidate":false,"about_ca_system_consensus":false,"about_ca_system_score_codex":0.0005384742,"about_ca_system_score_gemma":0.0006416411,"about_ca_topic_candidate":false,"about_ca_topic_consensus":false,"about_ca_topic_score_codex":0.0008191835,"about_ca_topic_score_gemma":0.001243207,"domain_scores_codex":[0.9996203,0.000160472,0.00002570495,0.00008597464,0.00004215778,0.00006531325],"domain_scores_gemma":[0.9994521,0.0001204558,0.0001312654,0.00003436278,0.00005102472,0.0002107981],"domain_codex":null,"domain_gemma":null,"domain_candidate":null,"domain_consensus":null,"study_design_codex":"randomized_trial","study_design_gemma":"randomized_trial","study_design_scores_codex":[0.9474184,0.008584331,0.001294112,0.0004443739,0.0009150168,0.00007769933,0.00009642523,0.0003689197,0.01841821,0.00006328403,0.0007485098,0.02157067],"study_design_scores_gemma":[0.7116744,0.2732649,0.01048993,0.0000417112,0.0009729609,0.00008861458,0.00005869961,0.0007115246,0.001581387,0.0001590071,0.000910131,0.00004671941],"study_design_candidate":"randomized_trial","study_design_consensus":"randomized_trial","genre_codex":"empirical","genre_gemma":"empirical","genre_scores_codex":[0.9947547,0.002734731,0.0003209686,0.0002175509,0.0001490046,0.0005830349,0.0004320227,0.00006947222,0.0007384218],"genre_scores_gemma":[0.9928868,0.001881698,0.0007508845,0.0007531088,0.0002698902,0.001076602,0.0006680477,0.00002465191,0.001688306],"genre_candidate":"empirical","genre_consensus":"empirical","teacher_disagreement_score":0.002734729,"threshold_uncertainty_score":0.009148538,"prediction_status":"machine_predicted_unvalidated"},"machine_scores":{"provisional":true,"baseline":true,"maturity_gate_passed":false,"score_opus":0.05197087178715996,"score_gpt":0.3354768177880474,"score_spread":0.2835059460008874,"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."}}