Enzyme replacement

What is Enzyme Replacement Therapy?

Enzyme Replacement Therapy (ERT) is a medical treatment used to treat diseases caused by deficiencies in certain enzymes. It works by replacing a defective enzyme with a new, functioning one. This works to help restore normal biochemical function to the affected cells, which can help to reduce or even eliminate some of the symptoms of the disease.

The treatment can be used in many different forms of disease, including metabolic diseases, musculoskeletal diseases, and neurodegenerative diseases. It is an important part of many medical treatments, providing effective relief and a sense of well-being to those suffering from these illnesses.

How Does Enzyme Replacement Therapy Work?

Enzyme Replacement Therapy works by introducing a new, functioning enzyme to replace the defective enzyme that is missing or not functioning properly. This is done either through administering the enzyme directly into the bloodstream or by attaching it to a viral vector that helps to distribute the enzyme throughout the body. From there, the enzyme can then begin to perform its functions, helping to restore normal biochemical processes.

The process can be repeated regularly, depending on the type of disease and the individual’s response to the treatment. Some patients may need multiple treatments in order to maintain the effectiveness of the therapy and/or to manage symptoms, while others may only need it once or twice a year.

Benefits of Enzyme Replacement Therapy

The primary benefit of Enzyme Replacement Therapy is that it can provide relief from the debilitating symptoms of certain enzyme deficiencies. Other benefits can include:

  • Improved energy levels
  • Reduced inflammation
  • Reduced risk of infection
  • Increased mobility/flexibility
  • Improved quality of life

In certain cases, Enzyme Replacement Therapy can even be used as an alternative to more aggressive treatments, such as surgery or medication. This treatment option can provide a more effective and natural course of action for some individuals suffering from certain enzyme deficiencies.


Enzyme Replacement Therapy is an important tool in the treatment of many medical conditions. By replacing defective enzymes, the therapy can help to improve the symptoms of a wide range of illnesses. In some cases, it can even provide an alternative to more invasive treatments, providing a safe and effective course of action for those who need it.