Glucagon-like peptide-1 analog therapy in rare genetic diseases: monogenic obesity, monogenic diabetes, and spinal muscular atrophy

• Published in: Acta diabetologica Vol. 60; no. 8; pp. 1099 - 1108
• Main Authors: Zaitoon, Hussein, Lubetzky, Ronit, Amir, Achiya Z., Moran-Lev, Hadar, Sagi, Liora, Yacobi-Bach, Michal, Borger, Ophir, Chorna, Efrat, Lebenthal, Yael, Brener, Avivit
• Format: Journal Article
• Language: English
• Published: Milan Springer Milan 01.08.2023; Springer Nature B.V
• Subjects: Age; Antihypertensives; Body mass index; Body weight gain; Comorbidity; Diabetes; Diabetes mellitus; Fatty liver; Genetic analysis; Genetic disorders; Glucagon; Glucagon-like peptide 1; Hypercholesterolemia; Internal Medicine; Literature reviews; Liver diseases; Medicine; Medicine & Public Health; Metabolic Diseases; Metformin; Mutation; Neuromuscular diseases; Obesity; Original Article; Peptides; Spinal muscular atrophy; Statins; Early-onset severe obesity; Childhood obesity; MC4R gene mutation; Hepatic nuclear factor 1 alpha (HNF1α MODY); Spinal muscular atrophy (SMA) type 3

Aim Implementing genetic analyses have unraveled rare alterations causing early-onset obesity and complications, in whom treatment is challenging. We aimed to report on the effects of adjuvant off-label therapy with liraglutide, glucagon-like peptide-1 analogue (GLP-1a), in rare genetic diagnoses. M...