2
Orphan Designations
1
FDA Approvals
11
Rare Diseases
0
News Articles
| Disease | Drug(s) | Designation | Approved |
|---|---|---|---|
| CHIME syndrome | Endari | Orphan Designation | - |
| bone marrow failure syndrome 3 | Endari | Orphan Designation | - |
| congenital short bowel syndrome | NutreStore | Orphan Designation | - |
| congenital short bowel syndrome, autosomal recessive | NutreStore | Orphan Designation | - |
| familial partial lipodystrophy, Dunnigan type | Endari | Orphan Designation | - |
| hyperphosphatasia with intellectual disability syndrome 6 | NutreStore | Orphan Designation | - |
| intestinal pseudoobstruction, neuronal, chronic idiopathic, X-linked | NutreStore | Orphan Designation | - |
| multiple congenital anomalies-hypotonia-seizures syndrome 2 | Endari | Orphan Designation | - |
| secondary short bowel syndrome | NutreStore | Orphan Designation | - |
| short bowel syndrome | NutreStore | Orphan Designation | - |
| sickle cell disease | Endari | Orphan Designation | - |