1
Orphan Designations
0
FDA Approvals
4
Rare Diseases
0
News Articles
| Disease | Drug(s) | Designation | Approved |
|---|---|---|---|
| hypotonia-failure to thrive-microcephaly syndrome | refanalin | Orphan Designation | - |
| insensitivity to pain with hyperplastic Myelinopathy | refanalin | Orphan Designation | - |
| teeth present at birth | refanalin | Orphan Designation | - |
| twin to twin transfusion syndrome | refanalin | Orphan Designation | - |