KEGG   DRUG: Tofersen
Entry
D11811                      Drug                                   
Name
Tofersen (JAN/USAN/INN);
BIIB067;
Qalsody (TN)
Product
QALSODY (Biogen)
Formula
C230H317N72O123P19S15
Exact mass
7123.1589
Mol weight
7127.90
Sequence
caggatacat ttctacagcu
  Type
Nucleotide
Remark
ATC code: N07XX22
Product: D11811<US>
Efficacy
Translation inhibitor
  Disease
Amyotrophic lateral sclerosis (SOD1 mutated) [DS:H00058]
  Type
Antisense oligonucleotide
Comment
Treatment of amyotrophic lateral sclerosis
Target
SOD1* [HSA_VAR:6647v1] [HSA:6647] [KO:K04565] (mRNA)
  Pathway
hsa04146  Peroxisome
hsa04213  Longevity regulating pathway - multiple species
hsa05014  Amyotrophic lateral sclerosis
hsa05022  Pathways of neurodegeneration - multiple diseases
  Network
nt06464  Amyotrophic lateral sclerosis
Brite
Anatomical Therapeutic Chemical (ATC) classification [BR:br08303]
 N NERVOUS SYSTEM
  N07 OTHER NERVOUS SYSTEM DRUGS
   N07X OTHER NERVOUS SYSTEM DRUGS
    N07XX Other nervous system drugs
     N07XX22 Tofersen
      D11811  Tofersen (JAN/USAN/INN) <US>
USP drug classification [BR:br08302]
 Central Nervous System Agents
  Amyotrophic Lateral Sclerosis (ALS) Agents
   Tofersen
    D11811  Tofersen (JAN/USAN/INN)
Target-based classification of drugs [BR:br08310]
 Enzymes
  Oxidoreductases (EC1)
   Superoxide dismutase
    SOD1* [HSA_VAR:6647v1]
     D11811  Tofersen (JAN/USAN/INN) <US>
New drug approvals in the USA [br08319.html]
 New molecular entities and new therapeutic biological products
  D11811
New drug approvals in Europe [br08329.html]
 European public assessment reports (EPAR) authorised medicine
  D11811
New drug approvals in the USA, Europe and Japan [br08328.html]
 Approval dates by FDA, EMA and PMDA
  D11811
Pharmacogenomic biomarkers [br08341.html]
 Germline mutations in genetic disorder treatments
  D11811
Other DBs
CAS: 2088232-70-4
PubChem: 405226676
LinkDB

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