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Agalsidase Beta: a review of its use in the management of Fabry disease.
Drugs. 2007; 67(3):435-55.D

Abstract

Agalsidase beta (Fabrazyme) is a recombinant human alpha-galactosidase A enzyme approved for intravenous use in the treatment of Fabry disease. Fabry disease is a progressive, multisystemic, potentially life threatening disorder caused by a deficiency of alpha-galactosidase A. This deficiency results in accumulation of glycosphingolipids, particularly globotriaosylceramide (GL-3), in the lysosomes of various tissues. This accumulation is the underlying driver of disease progression. Agalsidase beta provides an exogenous source of alpha-galactosidase A.Intravenous agalsidase beta is effective and well tolerated in patients with Fabry disease. In a phase III trial, agalsidase beta was shown to clear GL-3 from various target cells and, in a subsequent extension of this trial, prevent GL-3 reaccumulation. In a post-approval trial, agalsidase beta was shown to provide significant clinical benefit by reducing the risk of a major clinical event. Thus, agalsidase beta represents an important advance in the treatment of Fabry disease, and agalsidase beta therapy should be strongly considered in patients with Fabry disease who are suitable candidates.

Authors+Show Affiliations

Wolters Kluwer Health, Adis, Auckland, New Zealand. demail@adis.co.nzNo affiliation info available

Pub Type(s)

Journal Article
Review

Language

eng

PubMed ID

17335299

Citation

Keating, Gillian M., and Dene Simpson. "Agalsidase Beta: a Review of Its Use in the Management of Fabry Disease." Drugs, vol. 67, no. 3, 2007, pp. 435-55.
Keating GM, Simpson D. Agalsidase Beta: a review of its use in the management of Fabry disease. Drugs. 2007;67(3):435-55.
Keating, G. M., & Simpson, D. (2007). Agalsidase Beta: a review of its use in the management of Fabry disease. Drugs, 67(3), 435-55.
Keating GM, Simpson D. Agalsidase Beta: a Review of Its Use in the Management of Fabry Disease. Drugs. 2007;67(3):435-55. PubMed PMID: 17335299.
* Article titles in AMA citation format should be in sentence-case
TY - JOUR T1 - Agalsidase Beta: a review of its use in the management of Fabry disease. AU - Keating,Gillian M, AU - Simpson,Dene, PY - 2007/3/6/pubmed PY - 2007/5/16/medline PY - 2007/3/6/entrez SP - 435 EP - 55 JF - Drugs JO - Drugs VL - 67 IS - 3 N2 - Agalsidase beta (Fabrazyme) is a recombinant human alpha-galactosidase A enzyme approved for intravenous use in the treatment of Fabry disease. Fabry disease is a progressive, multisystemic, potentially life threatening disorder caused by a deficiency of alpha-galactosidase A. This deficiency results in accumulation of glycosphingolipids, particularly globotriaosylceramide (GL-3), in the lysosomes of various tissues. This accumulation is the underlying driver of disease progression. Agalsidase beta provides an exogenous source of alpha-galactosidase A.Intravenous agalsidase beta is effective and well tolerated in patients with Fabry disease. In a phase III trial, agalsidase beta was shown to clear GL-3 from various target cells and, in a subsequent extension of this trial, prevent GL-3 reaccumulation. In a post-approval trial, agalsidase beta was shown to provide significant clinical benefit by reducing the risk of a major clinical event. Thus, agalsidase beta represents an important advance in the treatment of Fabry disease, and agalsidase beta therapy should be strongly considered in patients with Fabry disease who are suitable candidates. SN - 0012-6667 UR - https://www.unboundmedicine.com/medline/citation/17335299/Agalsidase_Beta:_a_review_of_its_use_in_the_management_of_Fabry_disease_ L2 - https://dx.doi.org/10.2165/00003495-200767030-00007 DB - PRIME DP - Unbound Medicine ER -