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dose-dependent GI adverse events (nausea, vomiting) similar to other amylin analogues Exposure: Dose-proportional increases in Cmax and AUC Gastric Effects: Dose-dependent slowing of gastric emptying confirmed via paracetamol absorption test Appetite Suppression: Reduced ad libitum food intake at doses 1.2 mg Clinical Significance: Established feasibility of once-weekly dosing and identified dose range for Phase 2 studies

B12 also downregulated CSF2 ( P = 0.0071) and CXCL10 ( P = 0.0205) in leukocytes from impaired individuals, although these genes were not differentially regulated when compared to the non-impaired group

doi:10.1007/s40005-019-00436-0 108

In addition to improving accessibility and agility, DHTs also hold promising potential to improve therapeutic outcomes in rare diseases