2
Orphan Designations
0
FDA Approvals
6
Rare Diseases
0
News Articles
| Disease | Drug(s) | Designation | Approved |
|---|---|---|---|
| Hurler syndrome | Autologous CD34+ enriched cell population that contains hematopoietic stem and progenitor cells transduced ex-vivo using a lentiviral vector encoding alpha-L-iduronidase gene | Orphan Designation | - |
| Hurler-Scheie syndrome | Autologous CD34+ enriched cell population that contains hematopoietic stem and progenitor cells transduced ex-vivo using a lentiviral vector encoding alpha-L-iduronidase gene | Orphan Designation | - |
| McLeod neuroacanthocytosis syndrome | Autologous CD34+ enriched cell population that contains hematopoietic stem and progenitor cells transduced ex vivo using a lentiviral vector encoding the human gp91phox gene | Orphan Designation | - |
| Scheie syndrome | Autologous CD34+ enriched cell population that contains hematopoietic stem and progenitor cells transduced ex-vivo using a lentiviral vector encoding alpha-L-iduronidase gene | Orphan Designation | - |
| granulomatous disease, chronic, X-linked | Autologous CD34+ enriched cell population that contains hematopoietic stem and progenitor cells transduced ex vivo using a lentiviral vector encoding the human gp91phox gene | Orphan Designation | - |
| intestinal pseudoobstruction, neuronal, chronic idiopathic, X-linked | Autologous CD34+ enriched cell population that contains hematopoietic stem and progenitor cells transduced ex vivo using a lentiviral vector encoding the human gp91phox gene | Orphan Designation | - |