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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.


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