KEGG   DRUG: Onasemnogene abeparvovecHelp
Entry
D11559                      Drug                                   

Name
Onasemnogene abeparvovec (USAN/INN);
Onasemnogene abeparvovec-xioi;
Zolgensma (TN)
Efficacy
Spinal muscular atrophy treatment
  Disease
Spinal muscular atrophy (biallelic RPE65 mutations) [DS:H00455]
Comment
Gene therapy product
Target
SMN1 [HSA:6606] [KO:K13129]
Brite
Target-based classification of drugs [BR:br08310]
 Not elsewhere classified
  Signaling molecules
   Spliceosome
    SMN1
     D11559  Onasemnogene abeparvovec (USAN/INN)
New drug approvals in the USA [br08319.html]
 Cellular and gene therapy products
  D11559
New drug approvals in the USA, Europe and Japan [br08328.html]
 Approval dates by FDA, EMA and PMDA
  D11559
BRITE hierarchy
Other DBs
CAS: 1922968-73-7
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