Statements (19)
| Predicate | Object |
|---|---|
| gptkbp:instanceOf |
gene therapy
|
| gptkbp:activeIngredient |
gptkb:atidarsagene_autotemcel
|
| gptkbp:administeredBy |
autologous CD34+ hematopoietic stem cells transduced with a lentiviral vector
|
| gptkbp:approvalYear |
2020
|
| gptkbp:approvedBy |
gptkb:European_Union
gptkb:European_Medicines_Agency |
| gptkbp:ATCCode |
gptkb:V07AX28
|
| gptkbp:developer |
gptkb:Orchard_Therapeutics
|
| https://www.w3.org/2000/01/rdf-schema#label |
Libmeldy
|
| gptkbp:indication |
children with late infantile or early juvenile forms of metachromatic leukodystrophy
|
| gptkbp:legalStatus |
prescription only
|
| gptkbp:mechanismOfAction |
restores arylsulfatase A activity
|
| gptkbp:orphanDrugStatus |
yes
|
| gptkbp:routeOfAdministration |
intravenous
|
| gptkbp:synonym |
gptkb:OTL-200
gptkb:atidarsagene_autotemcel |
| gptkbp:usedFor |
gptkb:metachromatic_leukodystrophy
|
| gptkbp:bfsParent |
gptkb:Orchard_Therapeutics
|
| gptkbp:bfsLayer |
6
|