Authors' recommendations: X-linked hypophosphataemia is a rare, inherited condition that affects over 500 children in the UK. Phosphorous makes up an important part of bones and teeth. This condition stops the kidneys keeping enough phosphorous in the body for healthy bones and teeth. Symptoms include slow growth, bone deformities such as bowing of the legs, soft bones, pain in the legs and problems with the normal development of teeth. Some patients require surgery to help fix leg deformities. KRN23 is a new drug for the treatment of X-linked hypophosphataemia in children. It is injected under the skin. The only treatment currently available for this condition is phosphorus and vitamin D tablets, but these have to be taken several times a day, can have severe side effects, may be unpleasant to take and don't always return phosphorus levels to normal. If KRN23 is licensed for use in the UK, it could be a new treatment option for children with this condition that may improve quality of life and be more convenient than current treatments.

Project Status: Completed

Year Published: 2016

URL for published report: http://www.hsric.nihr.ac.uk/topics/krn23-for-x-linked-hypophosphataemia-in-children-aged-5-12-years-first-line/

English language abstract: An English language summary is available

Publication Type: Not Assigned

Country: England, United Kingdom

Organisation Name: NIHR Horizon Scanning Centre

Contact Address: The NIHR Horizon Scanning Centre, Department of Public Health, Epidemiology, and Biostatistics, School of Health and Population Sciences, University of Birmingham, 90 Vincent Drive, Edgbaston, Birmingham, B15 2SP. United Kingdom. Tel: +44 121 414 7831, Fax: +44 121 2269

Contact Name: c.packer@bham.ac.uk

Contact Email: c.packer@bham.ac.uk

Copyright: NIHR Horizon Scanning Research&Intelligence Centre (NIHR HSRIC)