5
Orphan Designations
0
FDA Approvals
18
Rare Diseases
0
News Articles
| Disease | Drug(s) | Designation | Approved |
|---|---|---|---|
| Behcet disease | Salagen | Orphan Designation | - |
| HELIX syndrome | Salagen | Orphan Designation | - |
| HSD10 mitochondrial disease | Etidronate disodium | Orphan Designation | - |
| Koolen-de Vries syndrome | Salagen | Orphan Designation | - |
| achalasia, familial esophageal | Salagen | Orphan Designation | - |
| acute intermittent porphyria | Salagen | Orphan Designation | - |
| dry eye syndrome | Salagen | Orphan Designation | - |
| epidermolysis bullosa simplex 1C, localized | Salagen | Orphan Designation | - |
| foodborne botulism | Salagen | Orphan Designation | - |
| humoral hypercalcemia of malignancy | Didronel | Orphan Designation | - |
| iatrogenic botulism | Salagen | Orphan Designation | - |
| liver disease, severe congenital | Etidronate disodium | Orphan Designation | - |
| oculorenocerebellar syndrome | Etidronate disodium | Orphan Designation | - |
| parathyroid gland carcinoma | Didronel | Orphan Designation | - |
| progeroid and marfanoid aspect-lipodystrophy syndrome | Salagen | Orphan Designation | - |
| somatostatinoma | Didronel | Orphan Designation | - |
| trichothiodystrophy 4, nonphotosensitive | Salagen | Orphan Designation | - |
| xeroderma pigmentosum group D | Salagen | Orphan Designation | - |