Article
Effectiveness and Safety of Setmelanotide in a Patient With a Heterozygous PCSK1 Deficiency.
Obesity (Silver Spring, Md.) - 1 Sept 2026
Lytvyak Ellina, Rattol Arshdeep, Grunvald Eduardo
Abstract excerpt
Setmelanotide, a melanocortin 4 receptor (MC4R) agonist, is a promising pharmacological treatment option for people with rare monogenic obesity conditions affecting the leptin-melanocortin signaling pathway, including proprotein convertase subtilisin/kexin type 1 (PCSK1) gene mutations. It has been studied in people with homozygous mutations causing a complete deficiency of PCSK1. We report the first case of a...
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- Humans
- Female
- Proprotein Convertase 1
- Adult
- Receptor, Melanocortin, Type 4
- Heterozygote
- alpha-MSH