2
Orphan Designations
0
FDA Approvals
7
Rare Diseases
0
News Articles
| Disease | Drug(s) | Designation | Approved |
|---|---|---|---|
| beta-amino acids, renal transport of | Arginine butyrate | Orphan Designation | - |
| beta-mannosidosis | IsobutyramideArginine butyrate | Orphan Designation | - |
| beta-thalassemia-X-linked thrombocytopenia syndrome | IsobutyramideArginine butyrate | Orphan Designation | - |
| hemoglobin C-beta-thalassemia syndrome | Isobutyramide | Orphan Designation | - |
| hemoglobin E-beta-thalassemia syndrome | Isobutyramide | Orphan Designation | - |
| hemoglobin Lepore-beta-thalassemia syndrome | Isobutyramide | Orphan Designation | - |
| sickle cell-beta-thalassemia disease syndrome | Isobutyramide | Orphan Designation | - |