4
Orphan Designations
0
FDA Approvals
8
Rare Diseases
0
News Articles
| Disease | Drug(s) | Designation | Approved |
|---|---|---|---|
| Wolfram syndrome 1 | autologous ex vivo expanded polyclonal CD4+CD25+CD1271o/-FOXP3+ Regulatory T cells | Orphan Designation | - |
| amyotrophic lateral sclerosis | Allogeneic motor neuron progenitor cells derived from human embryonic stem cells | Orphan Designation | - |
| cerebral arteriopathy, autosomal dominant, with subcortical infarcts and leukoencephalopathy, type 1 | autologous ex vivo expanded polyclonal CD4+CD25+CD1271o/-FOXP3+ Regulatory T cells | Orphan Designation | - |
| grade III prostatic intraepithelial neoplasia | Melanoma autologous dendritic cell vaccine | Orphan Designation | - |
| immune dysregulation-polyendocrinopathy-enteropathy-X-linked syndrome | autologous ex vivo expanded polyclonal CD4+CD25+CD1271o/-FOXP3+ Regulatory T cells | Orphan Designation | - |
| spinal muscular atrophy | late stage human motor neutron progenitors | Orphan Designation | - |
| squamous carcinoma in situ | Melanoma autologous dendritic cell vaccine | Orphan Designation | - |
| uterine cervix carcinoma in situ | Melanoma autologous dendritic cell vaccine | Orphan Designation | - |