Argente J, Haqq AM, Schorfheide JL, Touchot N, Huber C, Wiedemann U et al.
This analysis aimed to assess the efficacy of setmelanotide over 52 weeks in patients with Bardet-Biedl syndrome (BBS) compared with an external natural history cohort from the international Clinical Registry Investigating BBS (CRIBBS).Patients with BBS ≥ 6 years of ag...
Hypothalamic obesity (HO) is a rare, complex disorder characterized by disruption of brain pathways regulating energy intake, expenditure, autonomic function, and hormonal signaling. It occurs in rare monogenic obesity syndromes affecting central leptin-melanocortin pathways or can be acquired (a...
Bardet-Biedl syndrome (BBS) is a rare, autosomal recessive genetic disorder. Polydactyly, obesity, pigmentary retinal degeneration, intellectual disability, kidney abnormalities and hypogonadism are common features. We report an infant who presented with obesity, micropenis, polydactyly and syndr...
Peng J, Ou Y, Zhou M, Wang X, Zhang X, Long H et al.
Hypothalamic obesity (HO) is a disabling disease caused by central nervous system (CNS) damage due to neurosurgery, trauma, or tumors, especially in hypothalamus. The pathological mechanism of its neural circuits is still unclear, and there is currently no corresponding drug due to the complex et...
Özbay B, Jülke EM, List M, Nowicki M, Els-Heindl S, Immig K et al.
The regulation of appetite by pharmaceuticals has gained significant interest for the treatment of obesity and cachexia. The melanocortin 4 receptor (MC4R) and the ghrelin receptor (GhrR) are known to play a crucial role in the regulation of energy homeostasis. Thus, peptide ligands, which modula...
Journal of clinical research in pediatric endocrinology··PMID: 41537273
Müller HL
Patients diagnosed with craniopharyngioma often experience rapid and pronounced weight gain that can progress to severe hypothalamic obesity. This phenomenon is predominantly attributed to disruption of critical hypothalamic regulatory circuits, caused either by direct tumor infiltration or by tr...
Skin pigmentation by the endogenous pigment melanin is a highly coordinated process in which hormones play a crucial role. They are synthesized not only in classical endocrine organs but also in the skin itself, which acts as an independent endocrine organ. Among the endocrine target st...
Diabetology & metabolic syndrome··PMID: 41457234
Sun Y, Abed M, Sohouli MH
The melanocortin-4 receptor (MC4R) agonists have emerged as potential treatments for obesity, particularly in patients with rare genetic syndromes. However, their overall effects on obesity and cardiometabolic risk factors remain uncertain. To systematically evaluate the efficacy of MC4R agonists...
To compare the hazard of age-related macular degeneration (AMD) among nondiabetic, weight loss-eligible adults prescribed glucagon-like peptide-1 receptor agonists (GLP-1 RAs; semaglutide or liraglutide) versus other weight loss (OWL) pharmacotherapies (phentermine, orlistat, setmelanotide, phent...
Faccioli N, Poitou C, Georget M, Bertin F, Azar-Kolakez A, Carette C et al.
Pathogenic variants in five established leptin-melanocortin pathway genes (LEP, LEPR, MC4R, PCSK1, POMC) are associated with severe early-onset obesity and are targets for emerging treatments. However, these variants are rare in these patients, suggesting the involvement of additional genes inter...
Sexual dysfunction (SD) is a frequent and underrecognized complication of obesity, mediated by a complex interplay of hormonal, vascular, metabolic, and psychosocial pathways. Despite the established link between weight reduction and improved sexual health, the specific effects of new pharmacolog...
The bioactive peptide setmelanotide is a validated MC4R agonist, yet its clinical utility is constrained by poor aqueous solubility and dose-limiting, off-target hyperpigmentation. To overcome these dual liabilities, we executed a synergistic optimization strategy guided by detailed SAR investiga...
Obesity is the most prevalent condition in high-income nations, primarily associated with increased risk of diabetes, cardiovascular disease (CVD), hypertension, and hyperlipidemia. Lifestyle modifications are a key determinant in non-pharmacological management that includes a combination of nutr...
This case describes the resolution of refractory chronic idiopathic urticaria (CIU) with setmelanotide in a patient with Bardet-Biedl Syndrome (BBS). While setmelanotide is approved for syndromic obesity, this report highlights a novel anti-inflammatory effect potentially mediated by melanocortin...
Bardet-Biedl syndrome (BBS) and Alström syndrome (AS) are rare autosomal recessive ciliopathies characterized by severe multisystemic involvement, including metabolic, sensory, and developmental impairments. Both conditions result from primary cilia dysfunction, disrupting pathways such as l...
Clinical, cosmetic and investigational dermatology··PMID: 41281582
Tang J, Lv B, Liu L, Hu Y, Li K, Xu L et al.
Drug-induced skin pigmentary changes are an underrecognized yet clinically significant type of adverse event (AE). Despite growing awareness, labeling for many implicated drugs remains incomplete.To conduct a comprehensive pharmacovigilance analysis of the FDA Adverse Event Reporting System (FAER...
Müller HL, Tanaka T, Hasegawa T, Isojima T, Mori J, Kurosaki M et al.
Acquired hypothalamic obesity (aHO) presents as rapid, clinically relevant, and persistent weight gain due to hypothalamic damage, and leads to significant morbidity/mortality and decreased quality of life. Causes include craniopharyngioma and other space-occupying lesions, neurosurgical interven...
Following the diagnosis of craniopharyngioma, patients frequently experience rapid and excessive weight gain resulting in morbid hypothalamic obesity. This condition is commonly attributed to damage to hypothalamic structures caused either by the tumor itself or its treatment. Hypothalamic obesit...
To evaluate the risk of developing age-related cataract among nondiabetic patients who are overweight or obese prescribed glucagon-like peptide-1 receptor agonists (GLP-1RAs) compared to other weight loss drugs (OWLD).Retrospective cohort study using the TriNetX Global Collaborative Network.Overw...
Glucagon-like peptide-1 receptor agonists (GLP-1RAs) are increasingly used for weight loss, but their ocular effects in nondiabetic individuals remain unclear. Prior studies suggest GLP-1RA use reduced age-related macular degeneration (AMD) risk in patients with diabetes, but applicability to non...
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