Authors' objectives:

To summarise the current research evidence on iaronidase alfa for mucopolysaccharidosis I (MPS I disease).

Authors' recommendations: - Clinical impact: Iaronidase is an innovative treatment for people with MPS I disorders. As there is no current treatment the clinical impact could be significant. - Service impact: People with MPS I disorders are currently seen at 4 major centres in the UK. Those starting iaronidase will need to be seen and assessed by specialists before they are able to start therapy. - Patient issues: Although the patient group is small, knowledge of this treatment and the role it will play in the management of the disease is widespread. - Financial and overall NHS impact If all those with Hurler-Scheie and Scheie disorders are treated in the first year, the impact on the NHS will be 6.9M GBP. The number eligible for treatment may increase if the parents of those with Hurler's syndrome request therapy although its efficacy for this patient group has not been shown in trials.

Authors' methods: Overview

Project Status: Completed

Year Published: 2001

URL for published report: http://www.hsric.nihr.ac.uk/search

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: National Horizon Scanning Centre