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Article ; Online: Management of Monogenic and Syndromic Obesity.

Han, Joan C / Rasmussen, Marcus C / Forte, Alison R / Schrage, Stephanie B / Zafar, Sarah K / Haqq, Andrea M

Gastroenterology clinics of North America

2023  Volume 52, Issue 4, Page(s) 733–750

Abstract: Similar to the general population, lifestyle interventions focused on nutrition and physical activity form the foundation for treating obesity caused by rare genetic disorders. Additional therapies, including metreleptin and setmelanotide, that target ... ...

Abstract Similar to the general population, lifestyle interventions focused on nutrition and physical activity form the foundation for treating obesity caused by rare genetic disorders. Additional therapies, including metreleptin and setmelanotide, that target defects within the leptin signaling pathway can effectively synergize with lifestyle efforts to treat monogenic disorders of leptin, leptin receptor, proopiomelanocortin (POMC), and proprotein convertase subtilisin/kexin type 1 (PCSK1) and syndromic conditions, such as the ciliopathies Bardet-Biedl and Alström syndromes, whose pathophysiological mechanisms also converge on the leptin pathway. Investigational treatments for Prader-Willi syndrome target specific defects caused by reduced expression of paternally derived genes within the chromosome 15q region.
MeSH term(s) Humans ; Leptin/genetics ; Obesity/genetics ; Obesity/therapy ; Obesity/metabolism ; Prader-Willi Syndrome/genetics ; Prader-Willi Syndrome/therapy
Chemical Substances Leptin
Language English
Publishing date 2023-09-27
Publishing country United States
Document type Journal Article ; Review
ZDB-ID 92114-2
ISSN 1558-1942 ; 0889-8553
ISSN (online) 1558-1942
ISSN 0889-8553
DOI 10.1016/j.gtc.2023.08.005
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