Enzyme Replacement Therapy

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What is enzyme replacement therapy (ERT)?
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Enzyme replacement therapy is a medical treatment that replaces a missing or deficient enzyme in patients with certain metabolic disorders.
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Which conditions are commonly treated with enzyme replacement therapy?
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ERT is commonly used to treat lysosomal storage diseases like Gaucher disease, Fabry disease, and Pompe disease.
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How is enzyme replacement therapy administered?
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ERT is often administered intravenously, usually in a clinical setting under medical supervision.
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What are some challenges associated with enzyme replacement therapy?
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Challenges include immune reactions, high cost of treatment, and limited distribution to certain tissues.
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What role do enzymes play in the body?
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Enzymes are proteins that act as catalysts, speeding up chemical reactions necessary for cellular functions.
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How does ERT address enzyme deficiencies?
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ERT provides the missing enzyme directly, allowing normal metabolic processes to occur.
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Why can't enzyme replacement therapy permanently cure genetic disorders?
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ERT does not correct the underlying genetic mutation causing the enzyme deficiency.
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How is the efficacy of enzyme replacement therapy measured?
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Efficacy is measured by symptom improvement, biochemical markers, and quality of life assessments.
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What are potential side effects of enzyme replacement therapy?
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Side effects can include infusion-related reactions, such as rash, itching, and,in rare cases, severe allergic reactions.
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Can enzyme replacement therapy be used for all enzyme deficiencies?
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Not all enzyme deficiencies are treatable with ERT, as it depends on factors like enzyme stability and delivery to affected tissues.
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What advancements are being researched in enzyme replacement therapy?
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Researchers are exploring gene therapy and more targeted delivery mechanisms to improve ERT efficacy.
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How do patients typically respond to enzyme replacement therapy?
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Patient response varies; most see symptom relief, but some may have minimal or adverse reactions.
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What is the cost consideration for enzyme replacement therapy?
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ERT can be very expensive, often costing thousands of dollars per treatment session.
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How does enzyme replacement therapy compare with other therapies for metabolic disorders?
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ERT is often more immediate in symptom management but may need to be combined with other treatments for optimal care.
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What is a major limitation of current enzyme replacement therapies?
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A primary limitation is the inability to effectively deliver enzymes to reach the central nervous system.
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