Enzyme replacement therapy has become an important treatment option for several rare genetic disorders caused by missing or deficient enzymes. Recent advances have improved treatment effectiveness, safety, and patient convenience. Researchers continue to develop innovative delivery methods that help enzymes reach affected tissues more efficiently.
Enzyme replacement therapy (ERT) is a medical treatment used to replace enzymes that are missing or not functioning properly in the body. It is commonly used for certain inherited metabolic disorders where enzyme deficiencies lead to the buildup of harmful substances. Over the years, advancements in research and biotechnology have improved the effectiveness of ERT and expanded its potential applications.