VYONDYS 53TM is the brand name for golodirsen is the first treatment approved by the Food and Drug Administration (FDA) on December 12th, 2019 for a specific group of patients with Duchenne muscular dystrophy (DMD).
Frequently Asked Questions About VYONDYS 53TM
Is my son eligible for VYONDYS 53TM?
VYONDYS 53TM is for the treatment in patients with a confirmed mutation amenable to exon 53 skipping.
How does VYONDYS 53TM work?
VYONDYS 53TM is designed to mask exon 53 in the mRNA (a temporary copy of a gene used to make a protein) of the DMD gene so the protein synthesis machinery can skip this exon and piece together the remaining exons to make a smaller, but functional, dystrophin protein.
How is VYONDYS 53TM given to patients?
The exon-skipping therapy is given intravenously (through the veins) as a once-weekly injection that takes roughly an hour.
What is the benefit of VYONDYS 53TM?
Clinical trial results showed an observed statistically significant increase in dystrophin production in skeletal muscle of patients treated with VYONDYS 53TM, which is reasonably likely to predict clinical benefit for those patients.
Is VYONDYS 53TM covered by insurance?
Insurance coverage varies from plan to plan and it will require prior authorization. Depending on your insurance review process, prior authorization rate of acquisition may vary. A patient assistance program for those using VYONDYS 53TM, called SareptAssist, is available to help with treatment use and availability, and insurance and other financial concerns.