Article
Proopiomelanocortin Deficiency Treated with a Melanocortin-4 Receptor Agonist.
The New England journal of medicine - 21 Jul 2016
Kühnen Peter, Clément Karine, Wiegand Susanna, Blankenstein Oliver, Gottesdiener Keith, Martini Lea L, Mai Knut, Blume-Peytavi Ulrike, Grüters Annette, Krude Heiko
Abstract excerpt
Patients with rare defects in the gene encoding proopiomelanocortin (POMC) have extreme early-onset obesity, hyperphagia, hypopigmentation, and hypocortisolism, resulting from the lack of the proopiomelanocortin-derived peptides melanocyte-stimulating hormone and corticotropin. In such patients, adrenal insufficiency must be treated with hydrocortisone early in life. No effective pharmacologic treatments have...
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