1
Orphan Designations
0
FDA Approvals
12
Rare Diseases
0
News Articles
| Disease | Drug(s) | Designation | Approved |
|---|---|---|---|
| Adams-Oliver syndrome 1 | Bronchitol® | Orphan Designation | - |
| Angelman syndrome | Bronchitol® | Orphan Designation | - |
| DYRK1A-related intellectual disability syndrome | Bronchitol® | Orphan Designation | - |
| Noonan syndrome 1 | Bronchitol® | Orphan Designation | - |
| Opitz G/BBB syndrome | Bronchitol® | Orphan Designation | - |
| acroosteolysis dominant type | Bronchitol® | Orphan Designation | - |
| anosmia for isobutyric acid | Bronchitol® | Orphan Designation | - |
| double nail for fifth toe | Bronchitol® | Orphan Designation | - |
| hemoglobin--variants for which the chain carrying the mutation 1S unknown or uncertain | Bronchitol® | Orphan Designation | - |
| mitochondrial DNA depletion syndrome 13 | Bronchitol® | Orphan Designation | - |
| neuroacanthocytosis | Bronchitol® | Orphan Designation | - |
| syndromic X-linked intellectual disability 34 | Bronchitol® | Orphan Designation | - |