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.