KEGG   DRUG: Eteplirsen
Entry
D09900                      Drug                                   
Name
Eteplirsen (USAN/INN);
Exondys 51 (TN)
Product
EXONDYS 51 (Sarepta Therapeutics)
Formula
C364H569N177O122P30
Exact mass
10300.5890
Mol weight
10305.75
Sequence
[P-deoxy-P-(dimethylamino)
](2',3'-dideoxy-2',3'-imino-2',3'-seco) (2'a→5')
(C-m5U- C-C-A-A-C-A-m5U-C-A-A-G-G-A-A-G-A-m5U-G-G-C-A-m5U-m5U-m5U-C-m5U-A-G)
  Type
Nucleotide
Remark
ATC code: M09AX06
Product: D09900<US>
Efficacy
Translation inhibitor
  Disease
Duchenne muscular dystrophy (DMD variant amenable to exon 51 skipping) [DS:H01963]
  Type
Antisense oligonucleotide
Comment
Treatment of Duchenne muscular dystrophy
Target
DMD* [HSA_VAR:1756v1] [HSA:1756] [KO:K10366] (pre-mRNA exon51)
  Pathway
hsa04820  Cytoskeleton in muscle cells
  Network
nt06539  Cytoskeleton in muscle cells
Brite
Anatomical Therapeutic Chemical (ATC) classification [BR:br08303]
 M MUSCULO-SKELETAL SYSTEM
  M09 OTHER DRUGS FOR DISORDERS OF THE MUSCULO-SKELETAL SYSTEM
   M09A OTHER DRUGS FOR DISORDERS OF THE MUSCULO-SKELETAL SYSTEM
    M09AX Other drugs for disorders of the musculo-skeletal system
     M09AX06 Eteplirsen
      D09900  Eteplirsen (USAN/INN) <US>
USP drug classification [BR:br08302]
 Genetic, Enzyme, or Protein Disorder: Replacement, Modifiers, Treatment
  Eteplirsen
   D09900  Eteplirsen (USAN/INN)
Target-based classification of drugs [BR:br08310]
 Not elsewhere classified
  Cellular process
   Cytoskeleton
    DMD* [HSA_VAR:1756v1]
     D09900  Eteplirsen (USAN/INN) <US>
New drug approvals in the USA [br08319.html]
 New molecular entities and new therapeutic biological products
  D09900
New drug approvals in the USA, Europe and Japan [br08328.html]
 Approval dates by FDA, EMA and PMDA
  D09900
Pharmacogenomic biomarkers [br08341.html]
 Germline mutations in genetic disorder treatments
  D09900
Other DBs
CAS: 1173755-55-9
PubChem: 124490612
LinkDB

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KEGG   DRUG: Viltolarsen
Entry
D11528                      Drug                                   
Name
Viltolarsen (JAN/USAN);
Viltepso (TN)
Product
VILTEPSO (NS Pharma)
Formula
C244H381N113O88P20
Exact mass
6921.3564
Mol weight
6924.82
Sequence
RNA, [P-deoxy-P-(dimethylamino)
](2′,3′-dideoxy-2′,3′-imino-2′,3′-seco) (2′a→5′)
(C-C-m5U-C-C-G-G-m5U-m5U-C-m5U-G-A-A-G-G-m5U-G-m5U-m5U-C)
  Type
Nucleotide
Remark
Therapeutic category: 1900
ATC code: M09AX12
Product: D11528<JP/US>
Efficacy
Translation inhibitor
  Disease
Duchenne muscular dystrophy (amenable to exon 53 skipping mutated) [DS:H01963]
  Type
Antisense oligonucleotide
Comment
Treatment of Duchenne muscular dystrophy
Target
DMD* [HSA_VAR:1756v1] [HSA:1756] [KO:K10366] (pre-mRNA exon53)
  Pathway
hsa04820  Cytoskeleton in muscle cells
  Network
nt06539  Cytoskeleton in muscle cells
Brite
Anatomical Therapeutic Chemical (ATC) classification [BR:br08303]
 M MUSCULO-SKELETAL SYSTEM
  M09 OTHER DRUGS FOR DISORDERS OF THE MUSCULO-SKELETAL SYSTEM
   M09A OTHER DRUGS FOR DISORDERS OF THE MUSCULO-SKELETAL SYSTEM
    M09AX Other drugs for disorders of the musculo-skeletal system
     M09AX12 Viltolarsen
      D11528  Viltolarsen (JAN/USAN) <JP/US>
USP drug classification [BR:br08302]
 Genetic, Enzyme, or Protein Disorder: Replacement, Modifiers, Treatment
  Viltolarsen
   D11528  Viltolarsen (JAN/USAN)
Therapeutic category of drugs in Japan [BR:br08301]
 1  Agents affecting nervous system and sensory organs
  19  Other agents affecting nervous system and sensory organs
   190  Other agents affecting nervous system and sensory organs
    1900  Other agents affecting nervous system and sensory organs
     D11528  Viltolarsen (JAN/USAN)
Target-based classification of drugs [BR:br08310]
 Not elsewhere classified
  Cellular process
   Cytoskeleton
    DMD* [HSA_VAR:1756v1]
     D11528  Viltolarsen (JAN/USAN) <JP/US>
New drug approvals in the USA [br08319.html]
 New molecular entities and new therapeutic biological products
  D11528
New drug approvals in Japan [br08318.html]
 Drugs with new active ingredients
  D11528
New drug approvals in the USA, Europe and Japan [br08328.html]
 Approval dates by FDA, EMA and PMDA
  D11528
Pharmacogenomic biomarkers [br08341.html]
 Germline mutations in genetic disorder treatments
  D11528
Other DBs
CAS: 2055732-84-6
PubChem: 384585504
LinkDB

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KEGG   DRUG: Golodirsen
Entry
D11707                      Drug                                   
Name
Golodirsen (USAN/INN);
Vyondys 53 (TN)
Product
VYONDYS 53 (Sarepta Therapeutics)
Formula
C305H481N138O112P25
Exact mass
8642.9625
Mol weight
8647.29
Sequence
RNA, [P-deoxy-P-(dimethylamino)
](2′,3′-dideoxy-2′,3′-imino-2′,3′-seco) (2′a→5′)
(G-m5U-m5U-G-C-C-m5U-C-C-G-G-m5U-m5U-C-m5U-G-A-A-G-G-m5U-G-m5U-m5U-C)
, 5′-[P-[4-[[2-[2-(2-hydroxyethoxy)
ethoxy]ethoxy]carbonyl]-1-piperazinyl]-N,N-dimethylphosphonamidate]
  Type
Nucleotide
Remark
ATC code: M09AX08
Product: D11707<US>
Efficacy
Translation inhibitor
  Disease
Duchenne muscular dystrophy (DMD variant amenable to exon 53 skipping) [DS:H01963]
  Type
Antisense oligonucleotide
Target
DMD* [HSA_VAR:1756v1] [HSA:1756] [KO:K10366]
  Pathway
hsa04820  Cytoskeleton in muscle cells
  Network
nt06539  Cytoskeleton in muscle cells
Brite
Anatomical Therapeutic Chemical (ATC) classification [BR:br08303]
 M MUSCULO-SKELETAL SYSTEM
  M09 OTHER DRUGS FOR DISORDERS OF THE MUSCULO-SKELETAL SYSTEM
   M09A OTHER DRUGS FOR DISORDERS OF THE MUSCULO-SKELETAL SYSTEM
    M09AX Other drugs for disorders of the musculo-skeletal system
     M09AX08 Golodirsen
      D11707  Golodirsen (USAN/INN) <US>
USP drug classification [BR:br08302]
 Genetic, Enzyme, or Protein Disorder: Replacement, Modifiers, Treatment
  Golodirsen
   D11707  Golodirsen (USAN/INN)
Target-based classification of drugs [BR:br08310]
 Not elsewhere classified
  Cellular process
   Cytoskeleton
    DMD* [HSA_VAR:1756v1]
     D11707  Golodirsen (USAN/INN) <US>
New drug approvals in the USA [br08319.html]
 New molecular entities and new therapeutic biological products
  D11707
New drug approvals in the USA, Europe and Japan [br08328.html]
 Approval dates by FDA, EMA and PMDA
  D11707
Pharmacogenomic biomarkers [br08341.html]
 Germline mutations in genetic disorder treatments
  D11707
Other DBs
CAS: 1422959-91-8
PubChem: 405226573
LinkDB

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KEGG   DRUG: Casimersen
Entry
D11988                      Drug                                   
Name
Casimersen (USAN/INN);
Amondys 45 (TN)
Product
AMONDYS 45 (Sarepta Therapeutics)
Formula
C268H424N124O95P22
Exact mass
7580.6386
Mol weight
7584.44
Sequence
RNA, [P-deoxy-P-(dimethylamino)
](2′,3′-dideoxy-2′,3′-imino-2′,3′-seco) (2′a→5′)
(C-A-A-m5U-G-C-C-A-m5U-C-C-m5U-G-G-A-G-m5U-m5U-C-C-m5U-G) ,
5′-[P-[4-[[2-[2-(2-hydroxyethoxy)
ethoxy]ethoxy]carbonyl]-1-piperazinyl]-N,N-dimethylphosphonamidate]
  Type
Nucleotide
Remark
ATC code: M09AX13
Product: D11988<US>
Efficacy
Translation inhibitor
  Disease
Duchenne muscular dystrophy (DMD variant amenable to exon 45 skipping) [DS:H01963]
  Type
Antisense oligonucleotide
Comment
Treatment of Duchenne muscular dystrophy
Target
DMD* [HSA_VAR:1756v1] [HSA:1756] [KO:K10366] (pre-mRNA exon45)
  Pathway
hsa04820  Cytoskeleton in muscle cells
  Network
nt06539  Cytoskeleton in muscle cells
Brite
Anatomical Therapeutic Chemical (ATC) classification [BR:br08303]
 M MUSCULO-SKELETAL SYSTEM
  M09 OTHER DRUGS FOR DISORDERS OF THE MUSCULO-SKELETAL SYSTEM
   M09A OTHER DRUGS FOR DISORDERS OF THE MUSCULO-SKELETAL SYSTEM
    M09AX Other drugs for disorders of the musculo-skeletal system
     M09AX13 Casimersen
      D11988  Casimersen (USAN/INN) <US>
USP drug classification [BR:br08302]
 Genetic, Enzyme, or Protein Disorder: Replacement, Modifiers, Treatment
  Casimersen
   D11988  Casimersen (USAN/INN)
Target-based classification of drugs [BR:br08310]
 Not elsewhere classified
  Cellular process
   Cytoskeleton
    DMD* [HSA_VAR:1756v1]
     D11988  Casimersen (USAN/INN) <US>
New drug approvals in the USA [br08319.html]
 New molecular entities and new therapeutic biological products
  D11988
New drug approvals in the USA, Europe and Japan [br08328.html]
 Approval dates by FDA, EMA and PMDA
  D11988
Pharmacogenomic biomarkers [br08341.html]
 Germline mutations in genetic disorder treatments
  D11988
Other DBs
CAS: 1422958-19-7
PubChem: 497620401
LinkDB

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