Alpha glucosidase for people with Pompe's disease - horizon scanning review

Record ID 32002000531
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:
English language abstract: An English language summary is available
Publication Type: Not Assigned
Country: England, United Kingdom
MeSH Terms
  • alpha-Glucosidases
  • Glycogen Storage Disease Type II
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:
Contact Email:
Copyright: National Horizon Scanning Centre (NHSC)
This is a bibliographic record of a published health technology assessment from a member of INAHTA or other HTA producer. No evaluation of the quality of this assessment has been made for the HTA database.