Overall, we found no evidence to support clinical evaluation of liraglutide as a potential candidate for the treatment of ALS
These other disorders include VLCAD deficiency, CPT2 deficiency, and carnitine acylcarnitine translocase (CACT) deficiency, mitochondrial trifunctional protein (MTP) deficiency (this includes the primary defect in MTP due to pathogenic variants in the long-chain 3-hydroxyacyl-CoA dehydrogenase subunit encoding gene: HADHA), and urea cycle disorders
PBH is thought to be caused by an excessive glucagon-like peptide-1 (GLP-1) response leading to hypoglycemia and impaired quality of life
How strong is the evidence for using these two agents together
non-significant) in clinical decision-making and to provide more intuitive data for individualized counseling, we conducted an exploratory Bayesian NMA, intended as a PoC, which introduces the ROPE to translate heterogeneous clinical evidence into posterior probabilities of achieving or exceeding predefined clinical thresholds
The evolving framework under ICH S1B(R1) further emphasizes a modern, evidence-integrated approach to long-term safety evaluation
