Authors' objectives: Hunter syndrome is a rare disorder which generally only affects males. It is caused by a deficiency in an enzyme called iduronate-2-sulfatase. This enzyme is essential for breaking down molecules in the body called glycosaminoglycans. These molecules stay stored in cells in the body and cause damage to organs. This can slow mental and physical development. Intrathecal idursulfase is a new drug for the treatment of Hunter syndrome injected directly into the spinal canal once every month. A different formulation of this drug is already used, and is given as weekly injections into the bloodstream. However, this method of giving idursulfase does not slow the effects of Hunter syndrome on the brain. Some studies have suggested that intrathecal idursulfase may be helpful for patients with mild to moderate disease who have learning and behavioural difficulties. If intrathecal idursulfase is licensed for use in the UK, it could be a new treatment option for patients with Hunter syndrome who have learning difficulties.

Project Status: Completed

Year Published: 2016

URL for published report: http://www.hsric.nihr.ac.uk/topics/intrathecal-idursulfase-elaprase-for-hunter-syndrome-mucopolysaccharidosis-type-ii/

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)