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Burosumab treatment in adults with X-linked hypophosphataemia: 96-week patient-reported outcomes and ambulatory function from a randomised phase 3 trial and open-label extension.
Briot, Karine; Portale, Anthony A; Brandi, Maria Luisa; Carpenter, Thomas O; Cheong, Hae Ii; Cohen-Solal, Martine; Crowley, Rachel K; Eastell, Richard; Imanishi, Yasuo; Ing, Steven; Insogna, Karl; Ito, Nobuaki; Jan de Beur, Suzanne; Javaid, Muhammad K; Kamenicky, Peter; Keen, Richard; Kubota, Takuo; Lachmann, Robin H; Perwad, Farzana; Pitukcheewanont, Pisit; Ralston, Stuart H; Takeuchi, Yasuhiro; Tanaka, Hiroyuki; Weber, Thomas J; Yoo, Han-Wook; Nixon, Annabel; Nixon, Mark; Sun, Wei; Williams, Angela; Imel, Erik A.
Affiliation
  • Briot K; Department of Rheumatology, Hôpital Cochin, Paris, France karine.briot@aphp.fr.
  • Portale AA; Department of Pediatrics, Division of Nephrology, University of California, San Francisco, California, USA.
  • Brandi ML; FIRMO Foundation, Florence, Italy.
  • Carpenter TO; Department of Pediatrics and Department of Orthopedics and Rehabilitation, Yale School of Medicine, New Haven, Connecticut, USA.
  • Cheong HI; Department of Pediatrics, Seoul National University Children's Hospital, Seoul, Republic of Korea.
  • Cohen-Solal M; Department of Rheumatology, Hôpital Lariboisière, Paris, France.
  • Crowley RK; INSERM U1132 BIOSCAR, University of Paris, Paris, France.
  • Eastell R; Department of Endocrinology, St Vincent's University Hospital, Dublin, Ireland.
  • Imanishi Y; University College Dublin School of Medicine, Dublin, Ireland.
  • Ing S; Department of Oncology and Metabolism, The University of Sheffield, Sheffield, UK.
  • Insogna K; Department of Metabolism, Endocrinology and Molecular Medicine, Osaka City University Graduate School of Medicine, Osaka, Japan.
  • Ito N; Division of Endocrinology, Diabetes, and Metabolism, The Ohio State University Wexner Medical Center, Columbus, Ohio, USA.
  • Jan de Beur S; Department of Internal Medicine, Yale School of Medicine, New Haven, Connecticut, USA.
  • Javaid MK; Division of Nephrology and Endocrinology, The University of Tokyo Hospital, Tokyo, Japan.
  • Kamenicky P; Division of Endocrinology and Metabolism, Department of Medicine, Johns Hopkins University School of Medicine, Baltimore, Maryland, USA.
  • Keen R; Nuffield Department of Orthopaedics, Rheumatology and Musculoskeletal Sciences, National Institute for Health Research Oxford Biomedical Research Centre, University of Oxford, Oxford, UK.
  • Kubota T; Endocrine Physiology and Pathophysiology, INSERM, Paris-Saclay University, Paris, France.
  • Lachmann RH; Hospital Bicetre, Paris, France.
  • Perwad F; Metabolic Bone Disease Unit, Royal National Orthopaedic Hospital NHS Trust, London, UK.
  • Pitukcheewanont P; Department of Pediatrics, Osaka University Graduate School of Medicine, Osaka, Japan.
  • Ralston SH; Charles Dent Metabolic Unit, National Hospital for Neurology and Neurosurgery, London, UK.
  • Takeuchi Y; Department of Pediatrics, Division of Nephrology, University of California, San Francisco, California, USA.
  • Tanaka H; Department of Pediatric Endocrinology, Children's Hospital Los Angeles, University of Southern California Keck School of Medicine, Los Angeles, California, USA.
  • Weber TJ; Rheumatology and Bone Diseases Unit, Medical Research Council Institute of Genetics and Molecular Medicine, The University of Edinburgh, Edinburgh, UK.
  • Yoo HW; Endocrine Center, Toranomon Hospital, Tokyo, Japan.
  • Nixon A; Department of Pediatrics, Okayama Saiseikai General Hospital, Okayama, Japan.
  • Nixon M; Division of Endocrinology, Metabolism and Nutrition, Department of Medicine, Duke University Medical Center, Durham, North Carolina, USA.
  • Sun W; Department of Pediatrics, Asan Medical Center Children's Hospital, Seoul, Republic of Korea.
  • Williams A; Chilli Consultancy, Salisbury, UK.
  • Imel EA; Chilli Consultancy, Salisbury, UK.
RMD Open ; 7(3)2021 09.
Article de En | MEDLINE | ID: mdl-34548383

Texte intégral: 1 Collection: 01-internacional Base de données: MEDLINE Sujet principal: Rachitisme hypophosphatémique familial Type d'étude: Clinical_trials / Prognostic_studies Aspects: Patient_preference Limites: Adult / Humans Langue: En Journal: RMD Open Année: 2021 Type de document: Article Pays d'affiliation: France

Texte intégral: 1 Collection: 01-internacional Base de données: MEDLINE Sujet principal: Rachitisme hypophosphatémique familial Type d'étude: Clinical_trials / Prognostic_studies Aspects: Patient_preference Limites: Adult / Humans Langue: En Journal: RMD Open Année: 2021 Type de document: Article Pays d'affiliation: France