Laronidase
Canadian Coordinating Office for Health Technology Assessment
Record ID 32004000077
English, French
Authors' objectives:
To summarize the available information on the use of laronidase (Aldurazyme TM, manufactured by BioMarin Pharmaceutical Inc./Genzyme Corporation) to treat patients with Hurler and Hurler-Scheie disorders of mucopolysaccharidosis I.
Authors' recommendations:
Laronidase is the first enzyme replacement therapy marketed for use in the treatment of mucopolysaccharidosis I (MPS I). Given the lack of treatment alternatives for this debilitating disease, laronidase will be perceived as an attractive option. It is as yet uncertain, given the spectrum of severity in MPS I, where this agent will be of most value - especially in comparison to bone marrow transplant. It is assumed that central nervous system decline is not amenable to this treatment. The clinical data do not allow us to predict the overall effect of long-term use. Those who choose to adopt this potentially expensive technology will need to consider the future value of additional information from patient registries and post-marketing studies.
Authors' methods:
Overview
Details
Project Status:
Completed
URL for project:
https://www.ccohta.ca/
Year Published:
2004
English language abstract:
An English language summary is available
Publication Type:
Not Assigned
Country:
Canada
MeSH Terms
- Mucopolysaccharidoses
- Mucopolysaccharidosis I
Contact
Organisation Name:
Canadian Coordinating Office for Health Technology Assessment
Contact Address:
600-865 Carling Avenue, Ottawa, ON K1S 5S8 Canada. Tel: +1 613 226 2553, Fax: +1 613 226 5392;
Contact Name:
requests@cadth.ca
Contact Email:
requests@cadth.ca
Copyright:
Canadian Coordinating Office for Health Technology Assessment (CCOHTA)
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.