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 ?
In addition, it has an exclusive licensing agreement to commercialize Hunterase, an enzyme replacement therapy for the treatment of Hunter syndrome, developed by GC Pharma and marketed in more than ten countries worldwide.
Eight-year clinical outcomes of long-term enzyme replacement therapy for 884 children with Gaucher disease type 1.
Fabry disease: enzyme replacement therapy in [alpha]-galactosidase A deficient mice.
Successful pregnancy and lactation outcome in a patient with Gaucher disease receiving enzyme replacement therapy, and the subsequent distribution and excretion of imiglucerase in human breast milk.
The enzyme replacement therapy that Maura begins on Monday is her best hope.
Will began Enzyme Replacement Therapy into his body about five years ago.
But the support of doctors and timely treatment with Enzyme Replacement Therapy (yes the patients are getting ERT) has helped our family overcome this ordeal.
Dr Chris Hendriksz, a consultant at Birmingham Children's Hospital who specialises in Inherited Metabolic Disorders, said he believed the enzyme replacement therapy slows down progression of the disease.
The CUPID trial (Calcium Up-regulation by Percutaneous administration of gene therapy In cardiac Disease) is a randomized, double-blind, placebo-controlled study to assess the efficacy and safety of MYDICAR, a genetically targeted enzyme replacement therapy for advanced heart failure.
Tokyo, Dec 24, 2009 - (JCN Newswire) - A New Drug Application for the pancreatic enzyme replacement therapy agent for pancreatic exocrine insufficiency, SA-001 (API name: Pancrelipase) that Solvay Seiyaku K.
Enzyme replacement therapy has been successful in treating one form of lysosomal storage disease called Gaucher disease.

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