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Jordan, a real VILTEPSO patient and compensated spokesperson, and his mother, Laura. VILTEPSO is an exon-skipping therapy that has been granted FDA accelerated approval based on its demonstrated increase in dystrophin in DMD patients amenable to exon 53 skipping.
Most VILTEPSO patients choose to stay on treatment. Real-world claims data show that, of an eligible cohort of patients, 86% remained on VILTEPSO treatment after 12 months.
VILTEPSO is an FDA-approved exon 53-skipping therapy proven to help produce a shortened dystrophin protein containing essential functional portions.
Exon skipping is a therapeutic technique that “skips over” an exon next to deleted exon(s) in amenable patients with DMD.
In a clinical study, VILTEPSO was shown to significantly increase dystrophin production in skeletal muscles of DMD patients.
*In the pivotal 24-week study, the most common adverse reactions with VILTEPSO included upper respiratory tract infection, injection site reaction, cough, and pyrexia.
Explore VILTEPSO’s primary and
long-term clinical studies.
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