Product Description
VYONDYS 53 is an antisense oligonucleotide indicated for the treatment of Duchenne muscular dystrophy in patients who have a confirmed mutation of the DMD gene that is amenable to exon 53 skipping. VYONDYS 53 uses Sarepta’s proprietary phosphorodiamidate morpholino oligomer (PMO) chemistry and exon-skipping technology to bind to exon 53 of dystrophin pre-mRNA, resulting in exclusion, or “skipping,” of this exon during mRNA processing in patients with genetic mutations that are amenable to exon 53 skipping. Exon skipping is intended to allow for production of an internally truncated dystrophin protein.
How to use
Your doctor or nurse will give you this medicine. Kindly do not self administer.
Side Effects
- headache
- fever
- falls
- abdominal pain
- runny or stuffy nose
- cough
- vomiting
- nausea
Storage
Store in a refrigerator (2 - 8°C). Do not freeze.℃
0.0
0 Reviews