1
Orphan Designations
0
FDA Approvals
6
Rare Diseases
0
News Articles
| Disease | Drug(s) | Designation | Approved |
|---|---|---|---|
| hypotonia-failure to thrive-microcephaly syndrome | T4 endonuclease V, liposome encapsulated | Orphan Designation | - |
| insensitivity to pain with hyperplastic Myelinopathy | T4 endonuclease V, liposome encapsulated | Orphan Designation | - |
| linear skin defects with multiple congenital anomalies 2 | T4 endonuclease V, liposome encapsulated | Orphan Designation | - |
| linear skin defects with multiple congenital anomalies 3 | T4 endonuclease V, liposome encapsulated | Orphan Designation | - |
| recessive dystrophic epidermolysis bullosa-generalized other | T4 endonuclease V, liposome encapsulated | Orphan Designation | - |
| twin to twin transfusion syndrome | T4 endonuclease V, liposome encapsulated | Orphan Designation | - |