FOR PATIENTS + CAREGIVERS
HOW VILTEPSO WORKS
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.
Healthy Gene
The Duchenne muscular dystrophy (DMD) gene is made up of exons. These exons work together like building blocks to instruct the body on how to make full-length dystrophin protein.
DMD Gene Mutation
A mutation or deletion in the DMD gene may impact the way exons fit together. As you can see, the exons aren't able to connect, resulting in a lack of dystrophin production.
Exon 53 Skipping
VILTEPSO is designed to skip over exon 53. In this case, the orange block is skipped so that the green block can fit next to the blue one. In DMD patients amenable to exon 53 skipping, this can result in production of uninterrupted dystrophin.
Shortened Dystrophin
Now, when exon 53 is skipped, a shortened form of the dystrophin protein can be created. VILTEPSO is proven to help the body make a shortened form of the dystrophin protein.
When is a child’s exon deletion amenable to exon-skipping therapy?
For children diagnosed with DMD, a simple genetic test can help identify their specific mutation. The next step is to use their exon range to determine if they are eligible for (sometimes referred to as “amenable to”) VILTEPSO, or another exon-skipping therapy.
The Exon Finder tool can demonstrate how that works: Just select an exon range (based on the results of the genetic test we mentioned earlier), and we’ll tell you if exon-skipping therapy is an option.
Select a range from the options listed here:
Select a range from the options listed here:
3-50
4-50
5-50
6-50
9-50
3-52
4-52
5-52
6-52
9-52
10-50
11-50
13-50
14-50
15-50
16-50
17-50
19-50
10-52
11-52
13-52
14-52
15-52
16-52
17-52
19-52
21-50
23-50
24-50
25-50
26-50
27-50
28-50
29-50
21-52
23-52
24-52
25-52
26-52
27-52
28-52
29-52
30-50
31-50
32-50
33-50
34-50
35-50
36-50
37-50
38-50
39-50
30-52
31-52
32-52
33-52
34-52
35-52
36-52
37-52
38-52
39-52
40-50
41-50
42-50
43-50
45-50
47-50
48-50
49-50
40-52
41-52
42-52
43-52
45-52
47-52
48-52
49-52
50
52
52-58
52-61
52-63
52-64
52-66
52-76
52-77
50-52
54-58
54-61
54-63
54-64
54-66
54-76
54-77
This table is not intended to deliver a diagnosis or to replace any discussions with your doctor or genetic counselor.
Can’t find your range?
This child may not be eligible for exon-skipping therapy—consult a doctor for more information.
The selected range of exon deletions suggests this child may be eligible for treatment with VILTEPSO!
If your child has been diagnosed with DMD, speak with a doctor about VILTEPSO.
Email me this recommendation
VILTEPSO: an exon-skipping therapy option
- Designed to bind to and induce skipping of exon 53 of the dystrophin pre-mRNA, resulting in the production of a shortened dystrophin protein that contains essential functional portions
- Patients taking VILTEPSO showed a mean increase in dystrophin levels to 5.9% of normal by week 25 vs 0.6% of normal at baseline
- 100% of patients showed an increase in dystrophin levels
- VILTEPSO can be used to treat patients with DMD with an exon 52 deletion
This selected range of exon deletions suggests this child may be eligible for treatment with an exon-skipping therapy.
If your child has been diagnosed with DMD, speak with a doctor about potential treatment options.
What is exon-skipping?
- Exon skipping is a genetic technique that assists in “skipping over” an exon
- Exon skipping can be used to treat specific exon deletions
Sign up to find out more
about VILTEPSO
Take advantage of NS Support and links to helpful resources
At NS Support we are dedicated to being a committed partner to the families coping with DMD.
Interested in learning about clinical trials for Duchenne muscular dystrophy?
For general inquiries on DMD clinical trials with VILTEPSO, or if you’re interested in your child participating in future trials, email us at trialinfo@nspharma.com.
IMPACT ON DYSTROPHIN LEVELS
Does VILTEPSO impact dystrophin production?
For muscles to function effectively, they need a protein called dystrophin to be localized to the right part of the muscle cell.
VILTEPSO was shown to increase dystrophin production to nearly 6% of normal levels
VILTEPSO was studied in 16 ambulatory, or walking, boys aged 4 to younger 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.
100% of children treated with VILTEPSO in the clinical trial showed an increase in dystrophin levels
- The significant increase in dystrophin production with VILTEPSO was identified by a method called Western blot and verified by a highly sensitive measuring technique known as mass spectrometry
VILTEPSO significantly increased dystrophin production at week 25 compared with baseline
SAFETY
Safety profile evaluated in two 24-week clinical studies
Adverse reactions reported in ≥10% of DMD patients treated with VILTEPSO 80 mg/kg per week
*
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.
DOSING
Taking VILTEPSO
Q:
What is an infusion?
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
Q:
How much medication is in each dose of VILTEPSO?
Your healthcare provider will calculate the dose 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
Q:
How long is the infusion?
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
Remember, YOUR DOCTOR IS ALWAYS YOUR BEST RESOURCE for information about DMD and related treatment. Speak to your doctor about whether VILTEPSO may be right for your child.
