Question 1

What is enzyme replacement therapy (ERT)?

Accepted Answer

Enzyme replacement therapy is a treatment where a functional enzyme is delivered-typically by IV infusion-to individuals lacking a specific enzyme due to a genetic condition, helping restore essential metabolic functions.
Source: NCBI Bookshelf

Question 2

How does enzyme replacement therapy work?

Accepted Answer

ERT supplies a working form of the missing or deficient enzyme so that the body can break down substances that would otherwise accumulate. It’s usually given via regular intravenous infusions.
Source: National Gaucher Foundation

Question 3

What conditions require enzyme replacement therapy?

Accepted Answer

ERT is used to treat certain rare genetic lysosomal storage disorders, including Gaucher disease, Fabry disease, Pompe disease, and mucopolysaccharidoses (MPS types).
Source: Wikipedia overview summarizing FDA–authorized ERT indications

Question 4

Why would someone need enzyme replacement therapy?

Accepted Answer

People need ERT when their bodies do not produce enough of a vital enzyme, leading to the buildup of harmful substances in cells. ERT can help manage symptoms and improve quality of life in affected individuals.

Question 5

What is enzyme replacement therapy used for in Gaucher disease?

Accepted Answer

In Gaucher disease, ERT helps replace the missing glucocerebrosidase enzyme, preventing the accumulation of fatty substances in organs like the liver, spleen, and bone marrow. It can reduce symptoms and improve organ function.

Question 6

Is enzyme replacement therapy used in lysosomal storage disorders?

Accepted Answer

Yes, enzyme replacement therapy is widely used in lysosomal storage disorders. These conditions involve faulty enzymes in the lysosomes, and ERT provides functional enzymes to support proper cellular function.

Question 7

What is an example of enzyme replacement therapy?

Accepted Answer

A common ERT example is imiglucerase (Cerezyme), a recombinant glucocerebrosidase used in Gaucher disease, given via IV infusion.
Source: Medscape / FDA-approved imiglucerase history

Question 8

What are common side effects of enzyme replacement therapy?

Accepted Answer

Common side effects may include infusion-related reactions such as fever, rash, or fatigue. Some patients may develop antibodies to the infused enzymes. Side effects can vary depending on the specific therapy.

Question 9

How effective is enzyme replacement therapy for long-term disease management?

Accepted Answer

ERT can be effective in slowing disease progression and improving symptoms, especially when started early. However, it may not reverse all damage and often requires lifelong treatment for sustained benefits.

Question 10

What are therapeutic enzymes used for?

Accepted Answer

Therapeutic enzymes are used to support or replace natural enzymes in the body. They can be used in genetic disorders, digestive enzyme deficiencies, and some cancer therapies to aid metabolic functions or reduce inflammation.

Enzyme Replacement Therapy Medications