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 ?
Genzyme General, a division of Genzyme Corporation, is developing Fabrazyme (agalsidase beta), an enzyme replacement therapy for patients with Fabry disease.
Sollpura represents the next generation of enzyme replacement therapy for patients with EPI.
Xena, a pupil at Gilbertstone Primary School, in South Yardley, started the enzyme replacement therapy on February 1 under the watchful eye of her parents Kat and Fazalriaz.
Gaucher disease and bone: laboratory and skeletal mineral density variations during a long period of enzyme replacement therapy.
Lilly said the deal gives it a "promising entry into enzyme replacement therapy," an area the drugmaker does not currently cover.
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.
Creon/Kreon is the global market leader in pancreatic enzyme replacement therapy for pancreatic exocrine insufficiency due to cystic fibrosis, chronic pancreatitis, and pancreatectomy and other conditions.
Food and Drug Administration (FDA) Gastrointestinal Drugs Advisory Committee voted today to recommend non-approval of liprotamase, a non-porcine pancreatic enzyme replacement therapy (PERT), currently under FDA review for the treatment of exocrine pancreatic insufficiency (EPI).
Manufacturing support from the NHLBI SMARTT program will enable production of additional material that will be used to determine the safety and efficacy of rhLCAT enzyme replacement therapy for patients with familial LCAT deficiency - a potentially life-threatening illness for which there is no FDA-approved treatment.
a biopharmaceutical company focused on the discovery and development of innovative treatments for cardiovascular diseases, today announced that 12-month data from its Phase 2 CUPID clinical trial of MYDICAR demonstrated significant improvements in clinical outcomes and key disease markers in advanced heart failure patients treated with the genetically-targeted enzyme replacement therapy.
High Affinity ZC-701 Fusion Enzyme Replacement Therapy Demonstrates Superior Potency and Efficacy to Myozyme in Nonclinical Studies
Alnara's Pancreatic Enzyme Replacement Therapy, Liprotamase, Currently Under FDA Review

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