Iduronate-2-sulfatase for Hunter Syndrome - horizon scanning review
NHSC
Record ID 32005000238
English
Authors' objectives:
This study aims to assess the effectiveness of iduronate-2-sulfatase for Hunter Syndrome.
Authors' recommendations:
Recombinant human iduronate-2-sulfatase (I2S, idursulfase) is an enzyme replacement therapy for patients with Hunter Syndrome (mucopolysaccharidosis II, MPS II). Data from a conference poster presentation of a phase I/II trial in 12 patients demonstrated a dosedependent reduction in urinary glycosaminoglycan. After 1 year of I2S treatment, liver and spleen reduction were observed, with improvements in the 6-minute walk test and joint range of motion. A phase II/III randomised controlled trial has completed patient recruitment (n=96) and results are expected mid to end 2005.
Authors' methods:
Overview
Details
Project Status:
Completed
Year Published:
2005
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
MeSH Terms
- Mucopolysaccharidosis II
Contact
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)
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.