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Efficacy and safety of setmelanotide, a melanocortin-4 receptor agonist, in patients with Bardet-Biedl syndrome and Alström syndrome: a multicentre, randomised, double-blind, placebo-controlled, phase 3 trial with an open-label period.
Haqq, Andrea M; Chung, Wendy K; Dollfus, Hélène; Haws, Robert M; Martos-Moreno, Gabriel Á; Poitou, Christine; Yanovski, Jack A; Mittleman, Robert S; Yuan, Guojun; Forsythe, Elizabeth; Clément, Karine; Argente, Jesús.
Afiliação
  • Haqq AM; Division of Pediatric Endocrinology, University of Alberta, Edmonton, AB, Canada.
  • Chung WK; Department of Pediatrics, Division of Molecular Genetics, Columbia University, New York, NY, USA.
  • Dollfus H; Department of Medical Genetics, CARGO, Hôpitaux Universitaires de Strasbourg, Strasbourg, France.
  • Haws RM; Marshfield Clinic Research Institute, Marshfield, WI, USA.
  • Martos-Moreno GÁ; Department of Pediatrics and Pediatric Endocrinology, Universidad Autónoma de Madrid, Hospital Infantil Universitario Niño Jesús, CIBER Fisiopatología de la Obesidad y Nutrición (CIBEROBN), Instituto de Salud Carlos III, Madrid, Spain.
  • Poitou C; Department of Nutrition, Assistance Publique-Hôpitaux de Paris, Pitié-Salpêtrière Hospital, Paris, France; Sorbonne Université, INSERM, NutriOmics Research Unit, Paris, France.
  • Yanovski JA; Eunice Kennedy Shriver National Institute of Child Health and Human Development, National Institutes of Health, Bethesda, MD, USA.
  • Mittleman RS; Rhythm Pharmaceuticals, Boston, MA, USA.
  • Yuan G; Rhythm Pharmaceuticals, Boston, MA, USA.
  • Forsythe E; University College London, Great Ormond Street Institute of Child Health, London, UK.
  • Clément K; Department of Nutrition, Assistance Publique-Hôpitaux de Paris, Pitié-Salpêtrière Hospital, Paris, France; Sorbonne Université, INSERM, NutriOmics Research Unit, Paris, France.
  • Argente J; Division of Pediatric Endocrinology, University of Alberta, Edmonton, AB, Canada; IMDEA Food Institute, Madrid, Spain. Electronic address: jesus.argente@fundacionendo.org.
Lancet Diabetes Endocrinol ; 10(12): 859-868, 2022 12.
Article em En | MEDLINE | ID: mdl-36356613

Texto completo: 1 Coleções: 01-internacional Base de dados: MEDLINE Assunto principal: Síndrome de Bardet-Biedl / Síndrome de Alstrom Tipo de estudo: Clinical_trials Limite: Humans Idioma: En Revista: Lancet Diabetes Endocrinol Ano de publicação: 2022 Tipo de documento: Article País de afiliação: Canadá País de publicação: Reino Unido

Texto completo: 1 Coleções: 01-internacional Base de dados: MEDLINE Assunto principal: Síndrome de Bardet-Biedl / Síndrome de Alstrom Tipo de estudo: Clinical_trials Limite: Humans Idioma: En Revista: Lancet Diabetes Endocrinol Ano de publicação: 2022 Tipo de documento: Article País de afiliação: Canadá País de publicação: Reino Unido