Vyondys 53
| Chemical Name | golodirsen |
| Dosage Form | Injectable (intravenous; 50 mg/mL) |
| Drug Class | Oligonucleotides |
| System | Musculoskeletal |
| Company | Sarepta Therapeutics Inc. |
| Approval Year | 2019 |
Indication
- Vyondys 53 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.