8
Orphan Designations
1
FDA Approvals
15
Rare Diseases
0
News Articles
| Disease | Drug(s) | Designation | Approved |
|---|---|---|---|
| DYRK1A-related intellectual disability syndrome | SecrefloSynthetic human secretinSynthetic porcine secretin | Orphan Designation | - |
| Pearson syndrome | Chirostim | Orphan Designation | - |
| Shwachman-Diamond syndrome 1 | Chirostim | Orphan Designation | - |
| Wolf-Hirschhorn syndrome | Secreflo | Orphan Designation | - |
| anosmia for isobutyric acid | SecrefloSynthetic human secretinSynthetic porcine secretin | Orphan Designation | - |
| cardioacrofacial dysplasia 2 | Secreflo | Orphan Designation | - |
| double nail for fifth toe | Secreflo | Orphan Designation | - |
| gastrin-producing neuroendocrine tumor | SecrefloChirostim | Orphan Designation | - |
| hemoglobin--variants for which the chain carrying the mutation 1S unknown or uncertain | Secreflo | Orphan Designation | - |
| maturity-onset diabetes of the young type 8 | SecrefloChirostim | Orphan Designation | - |
| neuroacanthocytosis | SecrefloSynthetic human secretinSynthetic porcine secretin | Orphan Designation | - |
| neutropenia, severe congenital, 8, autosomal dominant | Chirostim | Orphan Designation | - |
| orofaciodigital syndrome I | Secreflo | Orphan Designation | - |
| paranasal sinus Schneiderian papilloma | Secreflo | Orphan Designation | - |
| renal cysts and diabetes syndrome | Chirostim | Orphan Designation | - |