Statements (19)
| Predicate | Object |
|---|---|
| gptkbp:instanceOf |
gptkb:drug
therapeutic agent |
| gptkbp:approvedBy |
gptkb:FDA
adult patients pediatric patients |
| gptkbp:category |
small molecule
antisense oligonucleotide |
| gptkbp:effect |
increases production of functional SMN protein
|
| gptkbp:example |
gptkb:nusinersen
gptkb:risdiplam branaplam |
| https://www.w3.org/2000/01/rdf-schema#label |
SMN2 gene splicing modifier
|
| gptkbp:mechanismOfAction |
modifies splicing of SMN2 pre-mRNA
|
| gptkbp:routeOfAdministration |
oral
intrathecal |
| gptkbp:target |
gptkb:SMN2_gene
|
| gptkbp:usedFor |
spinal muscular atrophy
|
| gptkbp:bfsParent |
gptkb:nusinersen
|
| gptkbp:bfsLayer |
7
|