Enzyme replacement therapy for rare diseases: a review of clinical efficacy
Canadian Agency for Drugs and Technologies in Health
Record ID 32011001513
English
Authors' recommendations:
Limited evidence for the treatment of selected liposomal storage disorders with enzyme replacement therapy suggests efficacy in some of these conditions. The rare occurrence of liposomal storage disorders poses a challenge for obtaining efficacy data.
Details
Project Status:
Completed
URL for project:
http://www.cadth.ca/media/pdf//htis/sept-2011/RC0300_Enzyme_RT_in_Rare_Diseases_%20final.pdf
Year Published:
2011
English language abstract:
An English language summary is available
Publication Type:
Not Assigned
Country:
Canada
MeSH Terms
- Enzyme Replacement Therapy
- Gaucher Disease
- Lysosomal Storage Diseases
- Mucopolysaccharidosis I
- Mucopolysaccharidosis II
Contact
Organisation Name:
Canadian Agency for Drugs and Technologies in Health
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 Agency for Drugs and Technologies in Health (CADTH)
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.