Authors' objectives:

To summarise the current research evidence on alpha glucosidase for people with Pompe's disease.

Authors' recommendations: - Clinical impact: There is no treatment for Pompe disease and in the infant form it is rapidly fatal. Any treatment that prolongs function and life will therefore have a major clinical impact. The role of alpha-glucosidase in the juvenile and adult forms of Pompe disease has yet to be established. Although currently known patient numbers are low in the UK compared to the Netherlands, the availability of a therapy is likely to increase registered cases. - Service impact: Initially there may be service re-organisation required as alpha-glucosidase is administered intravenously on a weekly basis. - Additional factors: Any treatment for the fatal infantile or juvenile disorder will be welcomed by professionals and families. - Financial and overall NHS impact: The financial impact on the NHS is very much dependent on the role that alpha-glucosidase will play in the long-term therapy for those with Pompe disease. If long-term benefits are anticipated then alpha-glucosidase administration may continue for many years increasing the financial impact.

Authors' methods: Overview

Project Status: Completed

Year Published: 2002

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 (NHSC)