Golodirsen
Sign in to saveGolodirsen, 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.
Wikidata facts
- Subclass of
- chemical compound
- Mass
- 8642.962530582005
Show 3 more facts
- isomeric SMILES
- 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
- chemical formula
- C₃₀₅H₄₈₁N₁₃₈O₁₁₂P₂₅
- canonical SMILES
- Cc1cn(C2CN(P(=O)(OCC3CNCC(n4ccc(N)nc4=O)O3)N(C)C)CC(COP(=O)(N(C)C)N3CC(COP(=O)(N(C)C)N4CC(COP(=O)(N(C)C)N5CC(COP(=O)(N(C)C)N6CC(COP(=O)(N(C)C)N7CC(COP(=O)(N(C)C)N8CC(COP(=O)(N(C)C)N9CC(COP(=O)(N(C)C)N%10CC(COP(=O)(N(C)C)N%11CC(COP(=O)(N(C)C)N%12CC(COP(=O)(N(C)C)N%13CC(COP(=O)(N(C)C)N%14CC(COP(=O)(N(C)C)N%15CC(COP(=O)(N(C)C)N%16CC(COP(=O)(N(C)C)N%17CC(COP(=O)(N(C)C)N%18CC(COP(=O)(N(C)C)N%19CC(COP(=O)(N(C)C)N%20CC(COP(=O)(N(C)C)N%21CC(COP(=O)(N(C)C)N%22CC(COP(=O)(N(C)C)N%23CC(COP(=O)(N(C)C)N%24CC(COP(=O)(N(C)C)N%25CC(COP(=O)(N(C)C)N%26CCN(C(=O)OCCOCCOCCO)CC%26)OC(n%26cnc%27c(=O)[nH]c(N)nc%27%26)C%25)OC(n%25cc(C)c(=O)[nH]c%25=O)C%24)OC(n%24cc(C)c(=O)[nH]c%24=O)C%23)OC(n%23cnc%24c(=O)[nH]c(N)nc%24%23)C%22)OC(n%22ccc(N)nc%22=O)C%21)OC(n%21ccc(N)nc%21=O)C%20)OC(n%20cc(C)c(=O)[nH]c%20=O)C%19)OC(n%19ccc(N)nc%19=O)C%18)OC(n%18ccc(N)nc%18=O)C%17)OC(n%17cnc%18c(=O)[nH]c(N)nc%18%17)C%16)OC(n%16cnc%17c(=O)[nH]c(N)nc%17%16)C%15)OC(n%15cc(C)c(=O)[nH]c%15=O)C%14)OC(n%14cc(C)c(=O)[nH]c%14=O)C%13)OC(n%13ccc(N)nc%13=O)C%12)OC(n%12cc(C)c(=O)[nH]c%12=O)C%11)OC(n%11cnc%12c(=O)[nH]c(N)nc%12%11)C%10)OC(n%10cnc%11c(N)ncnc%11%10)C9)OC(n9cnc%10c(N)ncnc%109)C8)OC(n8cnc9c(=O)[nH]c(N)nc98)C7)OC(n7cnc8c(=O)[nH]c(N)nc87)C6)OC(n6cc(C)c(=O)[nH]c6=O)C5)OC(n5cnc6c(=O)[nH]c(N)nc65)C4)OC(n4cc(C)c(=O)[nH]c4=O)C3)O2)c(=O)[nH]c1=O
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Encyclopedic overview
9 sectionsContents
- Medical uses
- Mechanism of action
- Adverse effects
- Pharmacology
- Pharmacokinetics
- Clinical benefits
- Society and culture
- History
- References
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.
Excerpted from Wikipedia’s “Golodirsen” article, available under the CC BY-SA 4.0 licence.