Quality of life outcomes in two phase 3 trials of setmelanotide in patients with obesity due to LEPR or POMC deficiency

Orphanet Journal of Rare Diseases

Peter Kühnen¹  Philipp M. Krabusch¹  Martin Wabitsch²  Julia von Schnurbein²  Patrick Callahan³  Christine Poitou⁴  Karine Clément⁴  Ari Gnanasakthy⁵  Costel Chirila⁵  Usha G. Mallya⁶  Murray Stewart⁶ 

[1] Charité - Universitätsmedizin Berlin, Corporate Member of Freie Universität Berlin und Humboldt-Universität zu Berlin, Institute for Experimental Pediatric Endocrinology, CVK, Augustenburger Platz 1, 13353, Berlin, Germany;Division of Pediatric Endocrinology and Diabetes, Center for Rare Endocrine Diseases, Department of Pediatrics and Adolescent Medicine, University of Ulm, Eythstraße 24, 89075, Ulm, Germany;Genesis Research, 111 River Street, Suite 1120, 07030, Hoboken, NJ, USA;Nutrition Department, Assistance Publique Hôpitaux de Paris, Pitié-Salpêtrière Hospital, IE3M Building, Room 637, 6th floor, 91 boulevard de l’Hôpital, 75013, Paris, France;INSERM, Nutrition and Obesity; Systemic Approaches (NutriOmique) Research Group, Sorbonne University, Room 637, 6th floor, 91 boulevard de l’Hôpital, 75013, Paris, France;RTI Health Solutions, P.O. Box 12194, 3040 East Cornwallis Road, 27709-2194, Research Triangle Park, NC, USA;Rhythm Pharmaceuticals, Inc., 222 Berkeley Street, Suite 1200, 02116, Boston, MA, USA;

关键词: Hyperphagia;    IWQOL-Lite;    PedsQL;    PHQ-9;    Rare genetic diseases of obesity;    Disease burden;    Melanocortin receptor;   

DOI  :  10.1186/s13023-022-02186-z

来源: Springer

【 授权许可】

CC BY