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As of March 2022, three medical drugs based on enzyme replacement therapy are available for Fabry disease: Agalsidase alfa, sold under the brand name Replagal by the company Takeda (since its acquisition of the company Shire), is a recombinant form of alpha-galactosidase A [21] It received approval in the EU in 2001. [22]
Pegunigalsidase alfa, sold under the brand name Elfabrio, is an enzyme replacement therapy for the treatment of Fabry disease. [2] [4] It is a recombinant human α-galactosidase-A. [4] It is a hydrolytic lysosomal neutral glycosphingolipid-specific enzyme. [2] The most common side effects are infusion-related reactions, hypersensitivity and ...
ERT is available for some lysosomal storage diseases: Gaucher disease, Fabry disease, MPS I, MPS II (Hunter syndrome), MPS VI and Pompe disease. [1] ERT does not correct the underlying genetic defect, but it increases the concentration of the enzyme that the patient is lacking. [1]
About Fabry Disease. Fabry disease is caused by a genetic deficiency of an enzyme known as alpha-galactosidase A or alpha-GAL, and is characterized by excessive accumulation of the lipid GL-3 in ...
In Fabry disease patients, 88% percent of patients develop IgG antibodies towards the injected recombinant enzyme, as it is foreign to their immune system. One suggested approach to solving this problem involves converting the paralogous enzyme α-NAGAL (NAGA) into one that has with α-GAL activity. Because patients still have a functional NAGA ...
Enzyme replacement therapy is available to treat mainly Fabry disease and Gaucher disease, and people with these types of sphingolipidoses may live well into adulthood. The other types are generally fatal by age 1 to 5 years for infantile forms, but progression may be mild for juvenile- or adult-onset forms.
Method for enhancing mutant enzyme activities in lysosomal storage disorders, (2003). [32] Chaperone-based therapy for Niemann-Pick disease, (2010). [33] Dose escalation enzyme replacement therapy for treating acid sphingomyelinase deficiency, (2013). [34] Dose escalation enzyme replacement therapy for treating acid sphingomyelinase deficiency ...
Enzyme replacement therapy is available mainly to treat Fabry disease and Gaucher disease and people with these types of sphingolipidoses may live well into adulthood. Generally, the other types are fatal by age 1 to 5 years for infantile forms, but progression may be mild for juvenile-onset or adult-onset forms.
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