3
Orphan Designations
0
FDA Approvals
12
Rare Diseases
0
News Articles
| Disease | Drug(s) | Designation | Approved |
|---|---|---|---|
| Angelman syndrome | Pentaspan | Orphan Designation | - |
| GM1 gangliosidosis | Selective inhibitor of polymorphonuclear leukocyte (PMN) elastase | Orphan Designation | - |
| Primrose syndrome | Pentaspan | Orphan Designation | - |
| acute sensory ataxic neuropathy | Pentaspan | Orphan Designation | - |
| anosmia for isobutyric acid | Trexan | Orphan Designation | - |
| aortic valve disease 1 | Pentaspan | Orphan Designation | - |
| aortic valve stenosis | Pentaspan | Orphan Designation | - |
| double nail for fifth toe | Trexan | Orphan Designation | - |
| hemoglobin--variants for which the chain carrying the mutation 1S unknown or uncertain | Trexan | Orphan Designation | - |
| intestinal pseudoobstruction, neuronal, chronic idiopathic, X-linked | Pentaspan | Orphan Designation | - |
| mucopolysaccharidosis-plus syndrome | Selective inhibitor of polymorphonuclear leukocyte (PMN) elastase | Orphan Designation | - |
| rhizomelic limb shortening with dysmorphic features | Selective inhibitor of polymorphonuclear leukocyte (PMN) elastase | Orphan Designation | - |