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
  • Cc1cn([C@H]2CN(P(=O)(OC[C@@H]3CN(P(=O)(OC[C@@H]4CN(P(=O)(OC[C@@H]5CN(P(=O)(OC[C@@H]6CN(P(=O)(OC[C@@H]7CN(P(=O)(OC[C@@H]8CN(P(=O)(OC[C@@H]9CN(P(=O)(OC[C@@H]%10CN(P(=O)(OC[C@@H]%11CN(P(=O)(OC[C@@H]%12CN(P(=O)(OC[C@@H]%13CN(P(=O)(OC[C@@H]%14CN(P(=O)(OC[C@@H]%15CN(P(=O)(OC[C@@H]%16CN(P(=O)(OC[C@@H]%17CN(P(=O)(OC[C@@H]%18CN(P(=O)(OC[C@@H]%19CN(P(=O)(OC[C@@H]%20CNC[C@H](n%21ccc(N)nc%21=O)O%20)N(C)C)C[C@H](n%20cc(C)c(=O)[nH]c%20=O)O%19)N(C)C)C[C@H](n%19cc(C)c(=O)[nH]c%19=O)O%18)N(C)C)C[C@H](n%18cnc%19c(=O)[nH]c(N)nc%19%18)O%17)N(C)C)C[C@H](n%17cc(C)c(=O)[nH]c%17=O)O%16)N(C)C)C[C@H](n%16cnc%17c(=O)[nH]c(N)nc%17%16)O%15)N(C)C)C[C@H](n%15cnc%16c(=O)[nH]c(N)nc%16%15)O%14)N(C)C)C[C@H](n%14cnc%15c(N)ncnc%15%14)O%13)N(C)C)C[C@H](n%13cnc%14c(N)ncnc%14%13)O%12)N(C)C)C[C@H](n%12cnc%13c(=O)[nH]c(N)nc%13%12)O%11)N(C)C)C[C@H](n%11cc(C)c(=O)[nH]c%11=O)O%10)N(C)C)C[C@H](n%10ccc(N)nc%10=O)O9)N(C)C)C[C@H](n9cc(C)c(=O)[nH]c9=O)O8)N(C)C)C[C@H](n8cc(C)c(=O)[nH]c8=O)O7)N(C)C)C[C@H](n7cnc8c(=O)[nH]c(N)nc87)O6)N(C)C)C[C@H](n6cnc7c(=O)[nH]c(N)nc76)O5)N(C)C)C[C@H](n5ccc(N)nc5=O)O4)N(C)C)C[C@H](n4ccc(N)nc4=O)O3)N(C)C)C[C@@H](COP(=O)(N(C)C)N3C[C@@H](COP(=O)(N(C)C)N4C[C@@H](COP(=O)(N(C)C)N5C[C@@H](COP(=O)(N(C)C)N6C[C@@H](COP(=O)(N(C)C)N7C[C@@H](COP(=O)(N(C)C)N8C[C@@H](COP(=O)(N(C)C)N9CCN(C(=O)OCCOCCOCCO)CC9)O[C@@H](n9cnc%10c(=O)[nH]c(N)nc%109)C8)O[C@@H](n8cc(C)c(=O)[nH]c8=O)C7)O[C@@H](n7cc(C)c(=O)[nH]c7=O)C6)O[C@@H](n6cnc7c(=O)[nH]c(N)nc76)C5)O[C@@H](n5ccc(N)nc5=O)C4)O[C@@H](n4ccc(N)nc4=O)C3)O2)c(=O)[nH]c1=O
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  • Key:KFINHGVWPSZMLV-CFTCKNMZSA-N

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

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

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