Golodirsen

Golodirsen
Clinical data
Trade namesVyondys 53
Other namesSRP-4053
AHFS/Drugs.comMonograph
License data
Routes of
administration
Intravenous
Drug classAntisense oligonucleotide
ATC code
Legal status
Legal status
Identifiers
CAS Number
DrugBank
UNII
KEGG
Chemical and physical data
FormulaC305H481N138O112P25
Molar mass8647.401 g·mol−1

Golodirsen, sold under the brand name Vyondys 53, is a medication used for the treatment of Duchenne muscular dystrophy.[2] It is an antisense oligonucleotide medication of phosphorodiamidate morpholino oligomer (PMO) chemistry.[1][3]

The most common side effects include headache, fever, fall, cough, vomiting, abdominal pain, cold symptoms (nasopharyngitis) and nausea.[2][1]

  1. ^ a b c "Vyondys 53- golodirsen injection". DailyMed. 31 March 2020. Archived from the original on 23 October 2020. Retrieved 6 August 2020.
  2. ^ a b Cite error: The named reference FDA PR was invoked but never defined (see the help page).
  3. ^ Anwar S, Yokota T (August 2020). "Golodirsen for Duchenne muscular dystrophy". Drugs of Today. 56 (8): 491–504. doi:10.1358/dot.2020.56.8.3159186. PMID 33025945. S2CID 222183389.