Patients & caregivers

How does VILTEPSO work?

VILTEPSO is an exon 53-skipping therapy that helps the body make its own dystrophin. To better understand how VILTEPSO works, imagine genes as being made up of building blocks called exons

Exon 53

Healthy Gene

The DMD gene is made up of exons. These exons work together, much like the building blocks above—connecting in a specific way to create clear instructions on how to make full-length dystrophin protein.

Exon 53 missing exon

DMD Gene Mutation

A mutation or deletion in the DMD gene may impact the way the building blocks, or certain exons, fit together. As the blocks above illustrate, the exons aren’t able to connect in the gene, which results in a lack of dystrophin production.

Skipped exon

Exon 53 Skipping

VILTEPSO is designed to skip over exon 53. In the image above, the orange block is skipped so the green block can fit next to the blue one. In DMD patients amenable to exon 53 skipping, this can result in the instructions for dystrophin production being communicated clearly.

Exon skipping 53

Shortened Dystrophin

As the blocks above show, when exon 53 is skipped, shortened dystrophin can be created. VILTEPSO is proven to help the body make a shortened form of the dystrophin protein.

How does VILTEPSO (80 mg/kg/wk) impact dystrophin production?

VILTEPSO was shown to increase dystrophin production to nearly 6% of normal levels

VILTEPSO was studied in 16 ambulatory, or walking, boys 4 to less than 10 years old who were receiving a stable dose of corticosteroids for at least 3 months.

Below, the average dystrophin levels after 20-24 weeks of treatment are compared with their average dystrophin levels before treatment.

Increased Dystrophin Graph 1 Increased Dystrophin Graph 1

100% of children treated with VILTEPSO in the clinical trial showed an increase in dystrophin levels

Increased Dystrophin Graph 2 Increased Dystrophin Graph 2

VILTEPSO safety overview

Adverse reactions reported in ≥10% of DMD patients treated with VILTEPSO 80 mg/kg once weekly

Safety graph Safety graph

*Upper respiratory tract infection includes the following terms: upper respiratory tract infection, nasopharyngitis, and rhinorrhea.

Injection site reaction includes the following terms: injection site bruising, injection site erythema, injection site reaction, and injection site swelling.


Because VILTEPSO is a once-weekly intravenous infusion, it may be helpful to know that it can be given by a healthcare professional at your home or at a treatment center.

You may have other questions or concerns about the dosing and administration of VILTEPSO:

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Q: What is an infusion?

A: An intravenous (IV) infusion goes into the bloodstream through a small needle and tube. It is a FAST way to get medication directly into the body

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Q: How much medication is in each VILTEPSO dosage?

A: Your healthcare provider will calculate the dosage based on your child’s body weight. 80 MILLIGRAMS of VILTEPSO is given for each kilogram (a kilogram is approximately 2.2 pounds) of your child’s weight per week

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Q: How long is the infusion?

A: The infusion lasts 60 MINUTES. But plan for some extra time before and after treatment in case you have questions for the nurse, or your child needs post-treatment observation

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VILTEPSO is indicated for the treatment of Duchenne muscular dystrophy (DMD) in patients who have a confirmed mutation of the DMD gene that is amenable to exon 53 skipping. This indication is approved under accelerated approval based on an increase in dystrophin production in skeletal muscle observed in patients treated with VILTEPSO. Continued approval for this indication may be contingent upon verification and description of clinical benefit in a confirmatory trial.

For more information about VILTEPSO, see full Prescribing Information.

Important Safety Information

  • In clinical studies, no patients experienced kidney toxicity during treatment with VILTEPSO. However, kidney toxicity from drugs like VILTEPSO may be possible. Your doctor may monitor the health of your kidneys before starting and during treatment with VILTEPSO.
  • Common side effects include upper respiratory tract infection, injection site reaction, cough, and fever.
  • These are not all of the possible side effects of VILTEPSO. For more information, ask your healthcare provider or pharmacist.
  • You are encouraged to report adverse events related to VILTEPSO. To do so, or for general inquiries, please call NS Pharma Medical Information at 1-866-NSPHARM (1-866-677-4276).

For more information about VILTEPSO, see full Prescribing Information.