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MC4R agonism promotes durable weight loss in patients with leptin receptor deficiency.
Clément, Karine; Biebermann, Heike; Farooqi, I Sadaf; Van der Ploeg, Lex; Wolters, Barbara; Poitou, Christine; Puder, Lia; Fiedorek, Fred; Gottesdiener, Keith; Kleinau, Gunnar; Heyder, Nicolas; Scheerer, Patrick; Blume-Peytavi, Ulrike; Jahnke, Irina; Sharma, Shubh; Mokrosinski, Jacek; Wiegand, Susanna; Müller, Anne; Weiß, Katja; Mai, Knut; Spranger, Joachim; Grüters, Annette; Blankenstein, Oliver; Krude, Heiko; Kühnen, Peter.
Affiliation
  • Clément K; Sorbonne Université, INSERM, NutriOmics team, Assistance Publique-Hôpitaux de Paris, Pitié-Salpêtrière Hospital, Paris, France.
  • Biebermann H; Institute for Experimental Pediatric Endocrinology, Charité - Universitätsmedizin Berlin, corporate member of Freie Universität Berlin, Humboldt-Universität zu Berlin and Berlin Institute of Health, Berlin, Germany.
  • Farooqi IS; University of Cambridge Metabolic Research Laboratories and NIHR Cambridge Biomedical Research Centre, Wellcome Trust-MRC Institute of Metabolic Science, Addenbrooke's Hospital, Cambridge, UK.
  • Van der Ploeg L; Rhythm Pharmaceuticals, Boston, MA, USA.
  • Wolters B; Institute for Experimental Pediatric Endocrinology, Charité - Universitätsmedizin Berlin, corporate member of Freie Universität Berlin, Humboldt-Universität zu Berlin and Berlin Institute of Health, Berlin, Germany.
  • Poitou C; Sorbonne Université, INSERM, NutriOmics team, Assistance Publique-Hôpitaux de Paris, Pitié-Salpêtrière Hospital, Paris, France.
  • Puder L; Institute for Experimental Pediatric Endocrinology, Charité - Universitätsmedizin Berlin, corporate member of Freie Universität Berlin, Humboldt-Universität zu Berlin and Berlin Institute of Health, Berlin, Germany.
  • Fiedorek F; Rhythm Pharmaceuticals, Boston, MA, USA.
  • Gottesdiener K; Rhythm Pharmaceuticals, Boston, MA, USA.
  • Kleinau G; Institute of Medical Physics and Biophysics, Group Protein X-ray Crystallography and Signal Transduction, Charité - Universitätsmedizin Berlin, corporate member of Freie Universität Berlin, Humboldt-Universität zu Berlin and Berlin Institute of Health, Berlin, Germany.
  • Heyder N; Institute of Medical Physics and Biophysics, Group Protein X-ray Crystallography and Signal Transduction, Charité - Universitätsmedizin Berlin, corporate member of Freie Universität Berlin, Humboldt-Universität zu Berlin and Berlin Institute of Health, Berlin, Germany.
  • Scheerer P; Institute of Medical Physics and Biophysics, Group Protein X-ray Crystallography and Signal Transduction, Charité - Universitätsmedizin Berlin, corporate member of Freie Universität Berlin, Humboldt-Universität zu Berlin and Berlin Institute of Health, Berlin, Germany.
  • Blume-Peytavi U; DZHK (German Centre for Cardiovascular Research), partner site Berlin, Berlin, Germany.
  • Jahnke I; Department of Dermatology and Allergy, Clinical Research Center for Hair and Skin Science, Charité - Universitätsmedizin Berlin, corporate member of Freie Universität Berlin, Humboldt-Universität zu Berlin and Berlin Institute of Health, Berlin, Germany.
  • Sharma S; Department of Dermatology and Allergy, Clinical Research Center for Hair and Skin Science, Charité - Universitätsmedizin Berlin, corporate member of Freie Universität Berlin, Humboldt-Universität zu Berlin and Berlin Institute of Health, Berlin, Germany.
  • Mokrosinski J; Rhythm Pharmaceuticals, Boston, MA, USA.
  • Wiegand S; University of Cambridge Metabolic Research Laboratories and NIHR Cambridge Biomedical Research Centre, Wellcome Trust-MRC Institute of Metabolic Science, Addenbrooke's Hospital, Cambridge, UK.
  • Müller A; Center for Chronic Sick Children, Charité - Universitätsmedizin Berlin, corporate member of Freie Universität Berlin, Humboldt-Universität zu Berlin and Berlin Institute of Health, Berlin, Germany.
  • Weiß K; Institute for Experimental Pediatric Endocrinology, Charité - Universitätsmedizin Berlin, corporate member of Freie Universität Berlin, Humboldt-Universität zu Berlin and Berlin Institute of Health, Berlin, Germany.
  • Mai K; Department of Pediatric Cardiology, Charité - Universitätsmedizin Berlin, corporate member of Freie Universität Berlin, Humboldt-Universität zu Berlin and Berlin Institute of Health, Berlin, Germany.
  • Spranger J; DZHK (German Centre for Cardiovascular Research), partner site Berlin, Berlin, Germany.
  • Grüters A; Department of Endocrinology, Diabetes, and Nutrition and Charité Center for Cardiovascular Research, Charité - Universitätsmedizin Berlin, corporate member of Freie Universität Berlin, Humboldt-Universität zu Berlin and Berlin Institute of Health, Berlin, Germany.
  • Blankenstein O; DZHK (German Centre for Cardiovascular Research), partner site Berlin, Berlin, Germany.
  • Krude H; Department of Endocrinology, Diabetes, and Nutrition and Charité Center for Cardiovascular Research, Charité - Universitätsmedizin Berlin, corporate member of Freie Universität Berlin, Humboldt-Universität zu Berlin and Berlin Institute of Health, Berlin, Germany.
  • Kühnen P; Department of Pediatric Endocrinology and Diabetes, Charité - Universitätsmedizin Berlin, corporate member of Freie Universität Berlin, Humboldt-Universität zu Berlin and Berlin Institute of Health, Berlin, Germany.
Nat Med ; 24(5): 551-555, 2018 05.
Article in En | MEDLINE | ID: mdl-29736023
Genetic defects underlying the melanocortin-4 receptor (MC4R) signaling pathway lead to severe obesity. Three severely obese LEPR-deficient individuals were administered the MC4R agonist setmelanotide, resulting in substantial and durable reductions in hyperphagia and body weight over an observation period of 45-61 weeks. Compared to formerly developed and tested MC4R agonists, setmelanotide has the unique capability of activating nuclear factor of activated T cell (NFAT) signaling and restoring function of this signaling pathway for selected MC4R variants. Our data demonstrate the potency of setmelanotide in treatment of individuals with diverse MC4R-related pathway deficiencies.
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Full text: 1 Collection: 01-internacional Database: MEDLINE Main subject: Weight Loss / Receptor, Melanocortin, Type 4 / Receptors, Leptin Limits: Adolescent / Adult / Humans / Male Language: En Journal: Nat Med Journal subject: BIOLOGIA MOLECULAR / MEDICINA Year: 2018 Document type: Article Affiliation country: Country of publication:
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Full text: 1 Collection: 01-internacional Database: MEDLINE Main subject: Weight Loss / Receptor, Melanocortin, Type 4 / Receptors, Leptin Limits: Adolescent / Adult / Humans / Male Language: En Journal: Nat Med Journal subject: BIOLOGIA MOLECULAR / MEDICINA Year: 2018 Document type: Article Affiliation country: Country of publication: