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Burosumab for the treatment of cutaneous-skeletal hypophosphatemia syndrome.
Abebe, Lillian; Phung, Kim; Robinson, Marie-Eve; Waldner, Richelle; Carsen, Sasha; Smit, Kevin; Tice, Andrew; Lazier, Joanna; Armour, Christine; Page, Marika; Dover, Saunya; Rauch, Frank; Koujok, Khaldoun; Ward, Leanne M.
Afiliação
  • Abebe L; The Ottawa Pediatric Bone Health Research Group, Children's Hospital of Eastern Ontario Research Institute, 401 Smyth Road, Ottawa, ON K1H 8L1, Canada.
  • Phung K; The Ottawa Pediatric Bone Health Research Group, Children's Hospital of Eastern Ontario Research Institute, 401 Smyth Road, Ottawa, ON K1H 8L1, Canada.
  • Robinson ME; Department of Pediatrics, Faculty of Medicine, University of Ottawa, 550 Cumberland St, Ottawa, ON K1N 6N5, Canada.
  • Waldner R; Department of Pediatrics, Faculty of Medicine, University of Ottawa, 550 Cumberland St, Ottawa, ON K1N 6N5, Canada.
  • Carsen S; Division of Endocrinology and Metabolism, Department of Pediatrics, Children's Hospital of Eastern Ontario, 401 Smyth Road, Ottawa, ON K1H 8L1, Canada.
  • Smit K; Department of Pediatrics, University of Alberta, 116 St & 85 Av, Edmonton, AB T6G 2R3, Canada.
  • Tice A; Department of Pediatrics, Faculty of Medicine, University of Ottawa, 550 Cumberland St, Ottawa, ON K1N 6N5, Canada.
  • Lazier J; Department of surgery, Children's Hospital of Eastern Ontario, 401 Smyth Road, Ottawa, ON K1H 8L1, Canada.
  • Armour C; Department of Pediatrics, Faculty of Medicine, University of Ottawa, 550 Cumberland St, Ottawa, ON K1N 6N5, Canada.
  • Page M; Department of surgery, Children's Hospital of Eastern Ontario, 401 Smyth Road, Ottawa, ON K1H 8L1, Canada.
  • Dover S; Department of Pediatrics, Faculty of Medicine, University of Ottawa, 550 Cumberland St, Ottawa, ON K1N 6N5, Canada.
  • Rauch F; Department of surgery, Children's Hospital of Eastern Ontario, 401 Smyth Road, Ottawa, ON K1H 8L1, Canada.
  • Koujok K; Department of Genetics, Children's Hospital of Eastern Ontario, 401 Smyth Road, Ottawa, ON K1H 8L1, Canada.
  • Ward LM; Department of Medical Genetics and Genomics, Faculty of Medicine, University of Ottawa, 550 Cumberland St, Ottawa, ON K1N 6N5, Canada.
Bone Rep ; 20: 101725, 2024 Mar.
Article em En | MEDLINE | ID: mdl-38229908
ABSTRACT
Cutaneous-skeletal hypophosphatemia syndrome (CSHS) is a rare bone disorder featuring fibroblast growth factor-23 (FGF23)-mediated hypophosphatemic rickets. We report a 2-year, 10-month-old girl with CSHS treated with burosumab, a novel human monoclonal antibody targeting FGF23. This approach was associated with rickets healing, improvement in growth and lower limb deformity, and clinically significant benefit to her functional mobility and motor development. This case report provides evidence for the effective use of FGF23-neutralizing antibody therapy beyond the classic FGF23-mediated disorders of X-linked hypophosphatemia and tumor-induced osteomalacia.
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Texto completo: 1 Coleções: 01-internacional Base de dados: MEDLINE Idioma: En Revista: Bone Rep Ano de publicação: 2024 Tipo de documento: Article País de afiliação: Canadá País de publicação: Estados Unidos
Texto completo
Adicionar na Minha BVS
Imprimir
XML
PubMed Links
Buscar no Google

Texto completo: 1 Coleções: 01-internacional Base de dados: MEDLINE Idioma: En Revista: Bone Rep Ano de publicação: 2024 Tipo de documento: Article País de afiliação: Canadá País de publicação: Estados Unidos