FOR PATIENTS + CAREGIVERS
HOW VILTEPSO WORKS
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.
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.
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.
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.
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 recommendationThis 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.
At NS Support we are dedicated to being a committed partner to the families coping with DMD.
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
For muscles to function effectively, they need a protein called dystrophin to be localized to the right part of the muscle cell.
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.
VILTEPSO significantly increased dystrophin production at week 25 compared with baseline
SAFETY
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Upper respiratory tract infection includes the following terms: upper respiratory tract infection, nasopharyngitis, and rhinorrhea.
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Injection site reaction includes the following terms: injection site bruising, injection site erythema, injection site reaction, and injection site swelling.
DOSING
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
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
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.
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.
For more information about VILTEPSO, see full Prescribing Information.