How does enzyme replacement therapy (ERT) work to treat Gaucher disease?

In treating Gaucher disease, enzyme replacement therapy (ERT) provides the enzyme that the body is missing. ERT helps the lysosomes chop up the waste material that has built up in the body's cells. ERT can help people begin to experience some relief of their symptoms. Some people get their ERT at the hospital and others can have it at home. It’s given through an intravenous drip or IV. The drugs are given through a peripheral line or a central access device such as a port-a-cath.

Continue Learning about Autosomal Recessive Genetic Disorders

Who is most at risk of autosomal recessive genetic disorders?
Honor Society of Nursing (STTI)Honor Society of Nursing (STTI)
Children who are born to parents who are both carriers of the same mutated autosomal recessive gene ...
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What is an autosomal recessive condition?
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In an autosomal recessive condition there is a mutation in both copies of a gene (the basic unit...
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What is lipoid proteinosis (LP)?
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Lipoid proteinosis (LP) is a rare disease that affects the skin and the brain. Three distinctive...
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What is the prognosis for lipoid proteinosis (LP)?
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Lipoid proteinosis (LP) has a stable or slowly progressive course. Children with LP may have behavio...
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