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Golodirsen

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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.

In the Vinony graph

Vinony's link graph records 30 inbound references to Golodirsen, and connects out to medical treatment, adult and intravenous infusion and defusion.

Vinony files it under Antisense RNA, Chemical articles having Jmol set and Chemical articles having Jmol set/None.

Vinony links it to 6 Wikipedia language editions.

Wikidata facts

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 sections
Contents
  • 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.

Available in 6 languages

via Wikidata sitelinks · CC0

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