Arylsulfatase B for mucopolysaccharidosis VI (Maroteaux-Lamy syndrome) - horizon scanning review
NHSC
Record ID 32005000244
English
Authors' objectives:
This study aims to assess the effectiveness of arylsulfatase B for mucopolysaccharidosis VI (Maroteaux-Lamy syndrome).
Authors' recommendations:
Arylsulfatase B is the first enzyme replacement therapy for mucopolysaccharidosis VI (MPS VI, Maroteaux-Lamy syndrome). A placebo-controlled, multi-centre phase III trial randomised 39 patients with MPS VI to arylsulfatase B or placebo. Patients who received arylsulfatase B at 24 weeks had reduced urinary glycosaminoglycans (GAGs) and walked on average 92 metres further compared with patients receiving placebo.
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
- N-Acetylgalactosamine-4-Sulfatase
- Mucopolysaccharidosis VI
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.