enzyme replacement therapy


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enzyme replacement therapy

n.
The administration of usually synthetic enzymes to limit or control the effects of a disease involving a specific enzyme deficiency, especially the use of pancreatic enzymes in the treatment of cystic fibrosis or other disorders of the pancreas, or the replacement of any of various enzymes implicated in disorders of lysosome storage.
References in periodicals archive ?
AGT(181, a novel, investigational enzyme replacement therapy for the treatment of both somatic and cognitive symptoms in patients with MPS I, was developed by re-engineering the enzyme iduronidase as fusion protein with an immunoglobulin G antibody targeting the insulin receptor.
This was because the children had no access to Enzyme Replacement Therapy,' she said.
The enzyme replacement therapy is designed to slow down the progression of the disease, and is being offered to five children through a "compassionate study" at London's Great Ormond Street Hospital.
In clinical studies, 67% of infants who received enzyme replacement therapy survived beyond 12 months of age, and children and adults had meaningful improvements in multiple disease-related liver and lipid abnormalities.
26) Babies born with the infantile-onset form of Pompe disease typically die within the first year of life, though enzyme replacement therapy can now prolong that lifespan.
Will began Enzyme Replacement Therapy into his body about five years ago.
Cambridge MA), a privately held biotechnology drug developer with an enzyme replacement therapy under review by the Food and Drug Administration.
a biopharmaceutical company focused on the discovery and development of innovative treatments for cardiovascular diseases, has announced that six-month data from its Phase 2 clinical trial of MYDICAR show improvements in clinical outcomes and disease markers in advanced heart failure patients treated with the genetically targeted enzyme replacement therapy.
Enzyme replacement therapy has been successful in treating one form of lysosomal storage disease called Gaucher disease.
Naglazyme was developed to supplement the deficient enzyme in MPS VI by providing an exogenous enzyme in a therapeutic approach known as enzyme replacement therapy (ERT).
The patients with CF had severely compromised exocrine pancreatic function, and all were receiving oral pancreatic enzyme replacement therapy consisting of 5000-10 000 U of porcine lipase x [kg.
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