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Essay / Research Paper Abstract
This 6 page paper considers th e nature of enzyme replacement therapy (ERT), which has been proposed for an increasing number of conditions, including Gaucher's disease, Fabry disease, different forms of hemophilia and cystic fibrosis. Bibliography lists 6 sources.
Page Count:
6 pages (~225 words per page)
File: MH11_MHEnzRep.rtf
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Unformatted sample text from the term paper:
considerable strides have been made in the use of ERT in the treatment of genetic disorders, one of the problematic elements relates to the varied modes of administration and the
impacts that ERT and intravenous treatments have on patients. In fact, site infections and reactions are a major issue when introducing ERT in new patient populations. In assessing the
modes of administration of enzyme replacement therapy (ERT), then, it is necessary to consider the methods for treatment relative to ERT use, the principles behind ERT and the specific nature
of conditions related to the use of ERT. In many cases (e.g. Gauchers disease, cystic fibrosis), the use of ERT in pediatric populations requires specific consideration of the impacts
of the modes of administration. ERT: An Overview Essentially, the term ERT, or enzyme replacement therapy, refers to the process of replacing a defective
or missing enzyme that are the result of a genetic disorder. Many genetic conditions result in physiological problems because of missing or defective enzymes and researchers have readily agreed
that the necessary therapeutic response is the replacement of the enzymes that are missing or defective. At the same time, a variety of outcomes have been noted in efforts
to replace missing or defective enzymes, and some have argued that treatment options can often cause problems that hinder the effectiveness of the treatment. Researchers have developed databases to study
the effectiveness of different ERT treatments and also determine the impacts of factors like mode of administration on effectiveness and quality of life. The Gaucher Registry (2004), for example,
is a database that provides documentation for the use of ERT in Gauchers disease. This database provides a comparative view of individuals who are not undergoing ERT and those
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