3
Orphan Designations
0
FDA Approvals
12
Rare Diseases
0
News Articles
| Disease | Drug(s) | Designation | Approved |
|---|---|---|---|
| Carney-Stratakis syndrome | Urogastrone | Orphan Designation | - |
| Hurler syndrome | Epidermal growth factor (human) | Orphan Designation | - |
| Khan-Khan-Katsanis syndrome | Fibronectin (plasma derived) | Orphan Designation | - |
| Sturge-Weber syndrome | Fibronectin (plasma derived) | Orphan Designation | - |
| anosmia for isobutyric acid | Fibronectin (plasma derived)Epidermal growth factor (human)Urogastrone | Orphan Designation | - |
| congenital hereditary endothelial dystrophy of cornea | Epidermal growth factor (human)Urogastrone | Orphan Designation | - |
| congenital non-bullous ichthyosiform erythroderma | Fibronectin (plasma derived) | Orphan Designation | - |
| double nail for fifth toe | Epidermal growth factor (human)Urogastrone | Orphan Designation | - |
| hemoglobin--variants for which the chain carrying the mutation 1S unknown or uncertain | Fibronectin (plasma derived) | Orphan Designation | - |
| mitochondrial DNA depletion syndrome 6 (hepatocerebral type) | Fibronectin (plasma derived) | Orphan Designation | - |
| modifier, X-linked, for Neurofunctional defects | Epidermal growth factor (human)Urogastrone | Orphan Designation | - |
| spatial visualization, aptitude for | Epidermal growth factor (human)Urogastrone | Orphan Designation | - |