Statements (23)
| Predicate | Object |
|---|---|
| gptkbp:instanceOf |
gene therapy
|
| gptkbp:administeredBy |
gptkb:Ospedale_San_Raffaele,_Milan,_Italy
intravenous infusion |
| gptkbp:alternativeTo |
hematopoietic stem cell transplantation
|
| gptkbp:approvalYear |
2016
|
| gptkbp:approvedBy |
gptkb:European_Medicines_Agency
|
| gptkbp:ATCCode |
not assigned
|
| gptkbp:contains |
autologous CD34+ cells transduced with retroviral vector
|
| gptkbp:developedBy |
gptkb:Fondazione_Telethon
gptkb:Ospedale_San_Raffaele GlaxoSmithKline |
| gptkbp:firstApprovedGeneTherapyFor |
gptkb:ADA-SCID
|
| https://www.w3.org/2000/01/rdf-schema#label |
Strimvelis
|
| gptkbp:indication |
patients with ADA-SCID lacking suitable HLA-matched donor
|
| gptkbp:marketedIn |
gptkb:European_Union
|
| gptkbp:mechanismOfAction |
restores adenosine deaminase activity
|
| gptkbp:orphanDrugStatus |
yes
|
| gptkbp:routeOfAdministration |
intravenous
|
| gptkbp:status |
approved
|
| gptkbp:treatment |
gptkb:ADA-SCID
|
| gptkbp:type |
ex vivo gene therapy
|
| gptkbp:bfsParent |
gptkb:gene_therapy
|
| gptkbp:bfsLayer |
5
|