What Is Enzyme Replacement Therapy?

Enzyme replacement therapy (ERT) is a non-invasive medical treatment that fulfills enzyme deficiencies in patients who suffer from chronic conditions. Mostly, ERTs are given through IV infusions through a controlled drip so the enzyme can flow into the bloodstream immediately.

The enzymes replace the ones that are absent in the body. These are derived from animal, human, and plant cells that are processed and genetically modified before they are administered. Once the patient receives these enzymes, the bodily functions inhibited by the deficiency resume working. However, the efficiency can vary from patient to patient, depending on their health.

How Does ERT Work?

ERT can treat several diseases, but for the sake of this guide, let’s take Pompe disease as an example of its efficiency. The genetic disorder is caused when the gene mutation triggers the production of acid alpha-glucosidase or GAA. While this enzyme typically breaks down glycogen, a sugar molecule that is then used as an energy source by the body, in Pompe, GAA levels can increase to toxic levels. This can lead to irreparable cell and muscle cell damage.

ERT for Pompe includes GAA enzyme replacement. A safe version of the enzyme to administer enters our cells to break down glycogen, thus preventing toxic buildup. Patients diagnosed with the condition should start enzyme therapy as soon as possible to curb the harmful symptoms.

Types of ERTs

There are two products that are currently approved for ERTs that treat Pompe disease, and Sanofi sells both. The doses are administered intravenously every other week. These include:


This product was the first enzyme approved for treating Pompe disease since 2010. It is also approved for the treatment of other diseases that can benefit from enzyme replacement in other countries but under a different brand name


This ERT solution is formulated to be administered directly to muscle cells, which is considered more efficient than Lumizyme. Nexviazyme is approved for US patients who have late-onset Pompe disease and are one year old or older. It is also approved in foreign markets under a different brand name.

While the ERTs cannot cure Pompe disease, they can slow its progression so patients can maintain a productive lifestyle. But the enzymes can be expensive for those not covered by health insurance. Many patients receive infusions at special infusion centers, but they have health insurance to cover the cost.

How the Rx Helper Can Help

The Rx Helper is a patient aid company that was formed to aid uninsured and underinsured patients in affording medication in the US. The free prescription aid program is meant to ease the stress and financial burden for patients who cannot afford critical prescriptions. While the medication is provided almost free, a nominal monthly fee is charged to cover the program’s cost. For more information about the benefits and program details, contact us today.



See If You Qualify For Prescription Assistance

Leave a Reply

Get In Touch

Call Us Now for a Free Consultation


Collect Free Consultation Service From Our Experts

By submitting your phone number on this website, you agree to the following terms and conditions:

  • You consent to receive SMS messages from The Rx Helper.
  • You understand that SMS messages may be sent to you regarding important updates about prescription assistance programs, medication reminders, and other relevant information related to healthcare services.
  • You may opt out of receiving SMS messages at any time by replying to any message with the word “STOP” or by visiting our website at https://www.therxhelper.com and clicking on the “Unsubscribe” link.
  • The Rx Helper will not share your phone number with any third parties without your consent.
  • You can review our privacy policy at [here] to learn more about how we collect, use, and protect your information.

Please note:

  • Message and data rates may apply.
  • We may use a variety of methods to deliver SMS messages, including short code and long code messaging.
  • We reserve the right to modify these terms and conditions at any time, so please check back periodically for updates.