Quality of life improvements following one year of setmelanotide in children and adult patients with Bardet–Biedl syndrome: phase 3 trial results

Orphanet Journal of Rare Diseases

Research

Karine Clément¹  Gabriel Á. Martos-Moreno²  Jesús Argente³  Andrea M. Haqq⁴  Elizabeth Forsythe⁵  Philip Beales⁵  Hélène Dollfus⁶  Robert M. Haws⁷  Costel Chirila⁸  Ari Gnanasakthy⁸  Usha G. Mallya⁹  Brieana C. Buckley⁹ 

[1] Assistance Publique-Hôpitaux de Paris, Nutrition Department, Pitié-Salpêtrière Hospital, Paris, France;Sorbonne Université, INSERM, NutriOmics Research Unit, Paris, France;Department of Pediatrics and Pediatric Endocrinology, Universidad Autónoma de Madrid, University Hospital Niño Jesús, Madrid, Spain;CIBER “Fisiopatología de la Obesidad y Nutrición” (CIBEROBN), Instituto de Salud Carlos III, Madrid, Spain;Department of Pediatrics and Pediatric Endocrinology, Universidad Autónoma de Madrid, University Hospital Niño Jesús, Madrid, Spain;CIBER “Fisiopatología de la Obesidad y Nutrición” (CIBEROBN), Instituto de Salud Carlos III, Madrid, Spain;IMDEA Food Institute, Madrid, Spain;Division of Pediatric Endocrinology, University of Alberta, 6-002E Li Ka Shing Centre for Health Research Innovation, T6G 2E1, Edmonton, AB, Canada;Genetics and Genomics Medicine Programme, University College London Great Ormond Street Institute of Child Health, London, UK;Hôpitaux Universitaires de Strasbourg, CARGO and Department of Medical Genetics, Strasbourg, France;Marshfield Clinic Research Institute, Marshfield, WI, USA;RTI Health Solutions, Research Triangle Park, NC, USA;Rhythm Pharmaceuticals, Inc., Boston, MA, USA;

关键词: BBS;    Genetic obesity;    IWQOL-Lite;    PedsQL;    Quality of life;    Setmelanotide;   

DOI  :  10.1186/s13023-022-02602-4

 received in 2022-08-15, accepted in 2022-12-19,  发布年份 2022

来源: Springer

【 授权许可】

CC BY   
© Rhythm Pharmaceuticals 2023

【 图 表 】

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Fig. 1