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Quality of life improvements following one year of setmelanotide in children and adult patients with Bardet-Biedl syndrome: phase 3 trial results.
Forsythe, Elizabeth; Haws, Robert M; Argente, Jesús; Beales, Philip; Martos-Moreno, Gabriel Á; Dollfus, Hélène; Chirila, Costel; Gnanasakthy, Ari; Buckley, Brieana C; Mallya, Usha G; Clément, Karine; Haqq, Andrea M.
Afiliación
  • Forsythe E; Genetics and Genomics Medicine Programme, University College London Great Ormond Street Institute of Child Health, London, UK.
  • Haws RM; Marshfield Clinic Research Institute, Marshfield, WI, USA.
  • Argente J; Department of Pediatrics and Pediatric Endocrinology, Universidad Autónoma de Madrid, University Hospital Niño Jesús, Madrid, Spain.
  • Beales P; CIBER "Fisiopatología de la Obesidad y Nutrición" (CIBEROBN), Instituto de Salud Carlos III, Madrid, Spain.
  • Martos-Moreno GÁ; IMDEA Food Institute, Madrid, Spain.
  • Dollfus H; Genetics and Genomics Medicine Programme, University College London Great Ormond Street Institute of Child Health, London, UK.
  • Chirila C; Department of Pediatrics and Pediatric Endocrinology, Universidad Autónoma de Madrid, University Hospital Niño Jesús, Madrid, Spain.
  • Gnanasakthy A; CIBER "Fisiopatología de la Obesidad y Nutrición" (CIBEROBN), Instituto de Salud Carlos III, Madrid, Spain.
  • Buckley BC; Hôpitaux Universitaires de Strasbourg, CARGO and Department of Medical Genetics, Strasbourg, France.
  • Mallya UG; RTI Health Solutions, Research Triangle Park, NC, USA.
  • Clément K; RTI Health Solutions, Research Triangle Park, NC, USA.
  • Haqq AM; Rhythm Pharmaceuticals, Inc., Boston, MA, USA.
Orphanet J Rare Dis ; 18(1): 12, 2023 01 16.
Article en En | MEDLINE | ID: mdl-36647077

Texto completo: 1 Base de datos: MEDLINE Asunto principal: Calidad de Vida / Síndrome de Bardet-Biedl Tipo de estudio: Prognostic_studies Idioma: En Revista: Orphanet J Rare Dis Asunto de la revista: MEDICINA Año: 2023 Tipo del documento: Article

Texto completo: 1 Base de datos: MEDLINE Asunto principal: Calidad de Vida / Síndrome de Bardet-Biedl Tipo de estudio: Prognostic_studies Idioma: En Revista: Orphanet J Rare Dis Asunto de la revista: MEDICINA Año: 2023 Tipo del documento: Article