This brand name is authorized in United States
The drug VYONDYS 53 contains one active pharmaceutical ingredient (API):
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Golodirsen
UNII 033072U4MZ - GOLODIRSEN
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Golodirsen is an antisense oligonucleotide of the phophorodiamidate morpholino oligomer (PMO) subclass. Golodirsen is designed to bind to exon 53 of dystrophin pre-mRNA resulting in exclusion of this exon during mRNA processing in patients with genetic mutations that are amenable to exon 53 skipping. Exon 53 skipping is intended to allow for production of an internally truncated dystrophin protein in patients with genetic mutations that are amenable to exon 53 skipping. |
Below package inserts are available for further reading:
| Title | Information Source | Document Type | |
|---|---|---|---|
| VYONDYS 53 Solution for injection | FDA, National Drug Code (US) | MPI, US: SPL/PLR |
This drug has been assigned below unique identifiers within the countries it is being marketed:
| Country | Identification scheme | Identifier(s) |
|---|---|---|
| Country: US | FDA, National Drug Code | Identifier(s): 60923-465 |
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