Gaucher enzyme replacement

Enzyme replacement Gaucher s disease, Fabry disease, and mucopolysaccharidosis Cerezyme, Fabrazyme, Aldurazyme... 

One example is an enzyme replacement drug used to treat a form of Gaucher disease, which is caused by an inherited error in the gene coding for the enzyme that breaks down the fatty substance cerebroside. In Gaucher disease, certain immune system cells... 

Brady, R.O., and N.W. Barton, Enzyme replacement therapy for Gaucher disease critical investigations beyond demonstration of clinical efficacy. Biochem Med Metab Biol, 1994.52(1) 1-9. 

Dosage form Ceredase is a sterile solution for injection, supplied in 5ml bottles containing alglucerase 80 units/ml. Cerezyme is supplied as a sterile lyophilized product for injection. Each vial contains imiglucerase 212 or 424 units. Amost all patients with Gaucher s disease use Cerezyme for enzyme replacement therapy. Ceredase is only available in limited supplies for the treatment of patients who do not tolerate Cerezyme. 

Long-term enzyme replacement therapy for patients with a confirmed diagnosis of type 1 Gaucher disease... 

General principles of enzyme replacement therapy for type 1 Gaucher disease can be developed at this time from the data presented in the preceding sections. Because the disease and its response to treatment are heterogeneous, the management of each patient most be individualized. At the outset of treatment, enzyme dosage should be selected based on the overall burden of disease and the... 

F. S. Furbish, H. R Blaii I. ShOoach, R G. Pentdiev, and R. O. Brody. Enzyme replacement therapy in Gaucher s disease large-scale purification of ghicocoe-bromdase suitable for human administration. Pmc. Nail Acad. ScL USA 74 3560... 

R. O. Brady, and N. W. Barton. Enzyme replacement therapy for Gaucher s disease skeletal responses to m croplw e-tarK ia l glucocerebimidase. Pediatrics... 

C. L. Vetderese, O. C. Graham, C. A. Holder-McShane, N. E Harnett, and N. W. Barton. Gaucher s disease a pilot study of the symptomatic responses to enzyme replacement therapy. I. Neurosci. Nurs. 25 296 (1993). 

B. Bembi, M. Zanatta, M. Carrozzi, F. Baralle, R. Gomati, B. Berra, and E Agosti. Enzyme replacement treatment in type i and type 3 Gaucher s disease, lancet 344 1679 0 994). 

A. Erikson, K. Johansson, J. E Mlnsson, and L. Svennerholm. Enzyme replacement therapy for infantile Gaucher disease Neumpediatrlcs 24 237 (1993). 

Parker RI, Barton NW, et al. Hematologic improvement in a patient with Gaucher disease on long term-enzyme replacement therapy evidence for decreased splenic sequestration and improved red blood cell survival. Am J Hematol 38 130-137. 

Macrophage mannose receptor binding is a particularly important issue in the assessment of rGCR as an enzyme replacement therapy because macrophages are the predominant cell type that accumulates lipid in Gaucher patients. In addition demonstrating that the recombinant protein behaved virtually identically to the naturally occurring protein would provide reassurance in the risk assessment process. 

Elstein D, Dweck A, Attias D, Hadas-Halpern I, Zevin S, Altarescu G, Aerts JF, van Weely S, Zimran A. Oral maintenance clinical trial with miglustat for type I Gaucher disease switch from or combination with intravenous enzyme replacement. Blood 2007 110 2296-2301. 

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