5
Orphan Designations
0
FDA Approvals
16
Rare Diseases
0
News Articles
| Disease | Drug(s) | Designation | Approved |
|---|---|---|---|
| acute graft versus host disease | Ryoncil | Orphan Designation | - |
| arthrogryposis, renal dysfunction, and cholestasis 1 | ex-vivo cultered adult human mesenchymal stem cells | Orphan Designation | - |
| hydrops-lactic acidosis-sideroblastic anemia-multisystemic failure syndrome | Human Allogeneic HLA-matched, Umbilical Cord Blood Hematopoietic Progenitor Cells Ex Vivo Expanded on Allogeneic Mesenchymal Precursor Cells | Orphan Designation | - |
| hypoplastic left heart syndrome | rexlemestrocel-L | Orphan Designation | - |
| idiopathic steroid-sensitive nephrotic syndrome with minimal change | Ryoncil | Orphan Designation | - |
| mitral valve insufficiency | Human Allogeneic HLA-matched, Umbilical Cord Blood Hematopoietic Progenitor Cells Ex Vivo Expanded on Allogeneic Mesenchymal Precursor Cells | Orphan Designation | - |
| post-thrombotic syndrome | rexlemestrocel-L | Orphan Designation | - |
| post-traumatic stress disorder | rexlemestrocel-L | Orphan Designation | - |
| post-vaccinal encephalitis | rexlemestrocel-L | Orphan Designation | - |
| spinal muscular atrophy with congenital bone fractures 1 | ex-vivo cultered adult human mesenchymal stem cells | Orphan Designation | - |
| steroid inherited metabolic disorder | Ryoncil | Orphan Designation | - |
| steroid lipomatosis | Ryoncil | Orphan Designation | - |
| steroid-resistant nephrotic syndrome | Ryoncil | Orphan Designation | - |
| steroid-responsive encephalopathy associated with autoimmune thyroiditis | Ryoncil | Orphan Designation | - |
| tricuspid valve insufficiency | Human Allogeneic HLA-matched, Umbilical Cord Blood Hematopoietic Progenitor Cells Ex Vivo Expanded on Allogeneic Mesenchymal Precursor Cells | Orphan Designation | - |
| type 1 diabetes mellitus | ex-vivo cultered adult human mesenchymal stem cells | Orphan Designation | - |