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Enzyme replacement therapy (ERT) is a medical treatment which replaces an enzyme that is deficient or absent in the body. [1] Usually, this is done by giving the patient an intravenous (IV) infusion of a solution containing the enzyme.
Cipaglucosidase alfa is a long-term enzyme replacement therapy used in combination with the enzyme stabilizer miglustat for the treatment of adults with late-onset Pompe disease (acid α-glucosidase [GAA] deficiency). [4] [5]
Enzymes can be classified by two main criteria: either amino acid sequence similarity (and thus evolutionary relationship) or enzymatic activity. Enzyme activity. An enzyme's name is often derived from its substrate or the chemical reaction it catalyzes, with the word ending in -ase.
GDEPT is a suicide gene therapy in which the enzyme required for prodrug conversion is produced within the target cell, using a gene delivered to it by gene therapy. When an adequate differential exists between the targeted cell and endogenous tissue, non-toxic prodrug is administered and is subsequently converted into its toxic form within the ...
Function: Amylase is an enzyme that is responsible for the breaking of the bonds in starches, polysaccharides, and complex carbohydrates to be turned into simple sugars that will be easier to absorb. Clinical Significance: Amylase also has medical history in the use of Pancreatic Enzyme Replacement Therapy (PERT). One of the components is ...
Substrate reduction therapy offers an approach to treatment of certain metabolic disorders, especially glycogen storage diseases and lysosomal storage disorders. In a storage disorder, a critical failure in a metabolic pathway prevents cellular breakdown and disposal of some large molecule. If residual breakdown through other pathways is ...
Pancreatic enzymes have been used as medications since at least the 1800s. [7] They are on the World Health Organization's List of Essential Medicines . [ 8 ] In 2022, it was the 253rd most commonly prescribed medication in the United States, with more than 1 million prescriptions.
Wobenzym, a combination of proteolytic enzymes and the antioxidant rutin, works systemically by targeting various tissues and organs in the body.Wobenzym is targeted at modulating the immune response to restore a healthy balance between anti-inflammatory and pro-inflammatory cytokines. [1]