1
Orphan Designations
0
FDA Approvals
3
Rare Diseases
0
News Articles
| Disease | Drug(s) | Designation | Approved |
|---|---|---|---|
| Dubowitz syndrome | Mesenchymal stem cell (MSC) derived small extracellular vesicles (EVs) loaded with short and modified interfering RNA (siRNA) against the phosphatase and tensin homolog (PTEN) protein | Orphan Designation | - |
| acute disseminated encephalomyelitis | Mesenchymal stem cell (MSC) derived small extracellular vesicles (EVs) loaded with short and modified interfering RNA (siRNA) against the phosphatase and tensin homolog (PTEN) protein | Orphan Designation | - |
| spinal muscular atrophy, distal, autosomal recessive, 6 | Mesenchymal stem cell (MSC) derived small extracellular vesicles (EVs) loaded with short and modified interfering RNA (siRNA) against the phosphatase and tensin homolog (PTEN) protein | Orphan Designation | - |