Posters
Presenting Author Academic/Professional Position
Elena Dike
Academic Level (Author 1)
Medical Student
Discipline Track
Clinical Science
Abstract Type
Research/Clinical
Abstract
Prader–Willi Syndrome (PWS) is a rare genetic disorder caused by loss of paternal expression in the 15q11–q13 region, with the SNORD116/SNHG14 cluster most strongly linked to hyperphagia. This hyperphagia is a major cause of morbidity in PWS. Vykat, a recently approved FDA drug to treat hyperphagia in PWS acts by opening KATP channels in pancreatic β-cells, thereby reducing insulin secretion and indirectly improving satiety. This presentation aims to highlight the central hypothalamic dysfunction from genetic imprinting defects and the peripheral action of Vykat. While Vykat represents a significant advance, especially for individuals with severe hyperphagia, it does not address the underlying genetic and hypothalamic pathology. Future pharmacologic approaches may need to focus on combining pharmacology with genetics to provide comprehensive disease management in PWS.
Presentation Type
Poster
Recommended Citation
Dike, Elena; Siddiqui, Nazia; Foamkom, Astrid-Ines; and Gadad, Bharathi, "Bridging Genetics and Pharmacology: Diazoxide Choline XR (Vykat) in the Treatment of Hyperphagia in Prader–Willi Syndrome" (2025). Research Colloquium. 38.
https://scholarworks.utrgv.edu/colloquium/2025/posters/38
Included in
Bridging Genetics and Pharmacology: Diazoxide Choline XR (Vykat) in the Treatment of Hyperphagia in Prader–Willi Syndrome
Prader–Willi Syndrome (PWS) is a rare genetic disorder caused by loss of paternal expression in the 15q11–q13 region, with the SNORD116/SNHG14 cluster most strongly linked to hyperphagia. This hyperphagia is a major cause of morbidity in PWS. Vykat, a recently approved FDA drug to treat hyperphagia in PWS acts by opening KATP channels in pancreatic β-cells, thereby reducing insulin secretion and indirectly improving satiety. This presentation aims to highlight the central hypothalamic dysfunction from genetic imprinting defects and the peripheral action of Vykat. While Vykat represents a significant advance, especially for individuals with severe hyperphagia, it does not address the underlying genetic and hypothalamic pathology. Future pharmacologic approaches may need to focus on combining pharmacology with genetics to provide comprehensive disease management in PWS.
