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 ?
Fabry disease: enzyme replacement therapy in [alpha]-galactosidase A deficient mice.
MYDICAR is a genetically targeted enzyme replacement therapy intended to restore levels of SERCA2a, a regulator of calcium cycling and contractility.
We also measured fecal elastase in samples from 5 patients with chronic pancreatitis before and during oral enzyme replacement therapy (500-1000 U of porcine lipase x [kg.
In October 2000, BioMarin initiated a randomized, double-blind Phase I clinical trial at Children's Hospital Oakland to test the safety of rhASB at two dose levels as an enzyme replacement therapy in six MPS-VI patients.
PHILADELPHIA -- Enzyme replacement therapy has conquered Fabry disease, a rare, lysosomal storage disease.
You and she should discuss the possibility of enzyme replacement therapy with her physician.
The US Food and Drug Administration (FDA) has granted approval to Ultragenyx Pharmaceutical's enzyme replacement therapy, Mepsevii (vestronidase alfa), intended for the treatment of Mucopolysaccharidosis VII (MPS VII, Sly syndrome) in both children and adults, it was reported yesterday.
Calias will have overall responsibility for the company's new Enzyme Replacement Therapy (ERT) programs, driving scientific milestones, enhancing the IP portfolio, overseeing new product development and contributing to the overall strategic direction of the company.
Calias will have overall responsibility for the Company s new Enzyme Replacement Therapy (ERT) programs, achieving the scientific milestones set in the business plan, enhancing the IP portfolio, overseeing new product development and contributing to the overall strategic direction of the Company.
His family were told his life expectancy would be around 11 or 12 but he started enzyme replacement therapy and his condition plateaued.
The merger, pending shareholder and regulatory nods, will enable Synageva to proceed with the development of its SBC-102 clinical programme, an enzyme replacement therapy for lysosomal acid lipase (LAL) deficiency.

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