Burosumab Therapy in Children with X-Linked Hypophosphatemia

Burosumab Therapy in Children with X-Linked Hypophosphatemia

In patients with X-linked hypophosphatemia, which is caused by PHEX mutations and is characterized by high FGF-23 and rickets, burosumab, an FGF-23 monoclonal antibody, improved renal phosphate reabsorption, serum phosphorus levels, and linear growth and reduced rickets severity.

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Veröffentlicht in:The New England journal of medicine 2018-05, Vol.378 (21), p.1987-1998
Hauptverfasser: Carpenter, Thomas O, Whyte, Michael P, Imel, Erik A, Boot, Annemieke M, Högler, Wolfgang, Linglart, Agnès, Padidela, Raja, van’t Hoff, William, Mao, Meng, Chen, Chao-Yin, Skrinar, Alison, Kakkis, Emil, San Martin, Javier, Portale, Anthony A
Format: Artikel
Sprache:eng
Schlagworte:
Alkaline phosphatase
Body weight
Children
Childrens health
Drug dosages
Fasting
Fibroblast growth factor 23
Hypophosphatemia
Metabolites
Monoclonal antibodies
Mutation
Osteomalacia
Pain
Pharmacodynamics
Phosphorus
Reabsorption
Rickets
Secretion
Vitamin D
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Beschreibung
Zusammenfassung:In patients with X-linked hypophosphatemia, which is caused by PHEX mutations and is characterized by high FGF-23 and rickets, burosumab, an FGF-23 monoclonal antibody, improved renal phosphate reabsorption, serum phosphorus levels, and linear growth and reduced rickets severity.
ISSN:0028-4793
1533-4406
DOI:10.1056/NEJMoa1714641