enzyme replacement therapy

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enzyme replacement therapy

A generic term for therapeutic administration of a congenitally defective or absent enzyme, which may be adminstered:  
• Directly, by coupling the enzyme to a carrier molecule or by organ transplantation; or  
• Indirectly, by introducing the gene into the recipient.

enzyme replacement therapy

Administration of enzymes to patients with congenital or acquired enzyme deficiency diseases, e.g., oral pancreatic enzymes to patients with chronic pancreatitis, or infused enzymes to patients with genetic disorders in which a single enzyme is lacking, such as galactosidase to patients with Fabry disease.

enzyme replacement therapy

Treatment of genetic diseases in which the disorder is the result of biochemical dysfunction resulting from the absence of a particular enzyme. Enzymes can now be made by recombinant DNA technology and have to be given by intravenous injection. The method has been used successfully to treat Gaucher's disease, a sphingolipidosis caused by the absence of the lysosomal enzyme glucocerebrosidase.
References in periodicals archive ?
But she does not want to risk waiting if she can help it and there is an enzyme replacement treatment, not available on the NHS, but available in America which she wants Brooklyn to have a chance at.
We are excited about this collaboration, which represents a significant step towards bringing, for the first time, a plant-based enzyme replacement treatment option to patients affected by Gaucher's disease," commented Dr.
announces that a new stem cell gene and enzyme replacement treatment has been performed on a fourteen month old child with Tay-Sachs disease on November 2, 2010.
Idursulfase, marketed by Shire Human Genetics Therapies, is the first and only enzyme replacement treatment for people suffering from Hunter syndrome (Mucopolysaccharidosis II) since this condition was identified 100 years ago.
American firm Biomarin Pharmaceuticals has claimed early success with a revolutionary enzyme replacement treatment in tests in the States.
Karl has a rare condition called Hunters Syndrome, that requires a weekly enzyme replacement treatment.
Food & Drug Administration (FDA) to issue its decision for VPRIVTM (velaglucerase alfa for injection), Shire's investigational enzyme replacement treatment for Type 1 Gaucher disease, by February 28.
recalled four batches of Fabrazyme, an enzyme replacement treatment for Fabry disease, in Europe because vials were incompletely filled, regulators said.
Barry Byrne, Director of the University of Florida Powell Center for Rare Disease Research and a leading authority on Pompe disease and related diseases, and a pioneer in developing new therapies to restore muscle function in Pompe and other inherited myopathies, noted, "Callidus' approach opens up a new avenue for improving drug targeting of enzyme replacement treatments for Lysosomal Storage Disorders.

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