1
Orphan Designations
0
FDA Approvals
10
Rare Diseases
0
News Articles
| Disease | Drug(s) | Designation | Approved |
|---|---|---|---|
| arrhythmogenic cardiomyopathy with wooly hair and keratoderma | VYNDAQEL; VYNDAMAX | Orphan Designation | - |
| histiocytoid cardiomyopathy | VYNDAQEL; VYNDAMAX | Orphan Designation | - |
| intellectual disability, autosomal dominant 15 | VYNDAQEL; VYNDAMAX | Orphan Designation | - |
| microcephaly-cardiomyopathy syndrome | VYNDAQEL; VYNDAMAX | Orphan Designation | - |
| myofibrillar myopathy 2 | VYNDAQEL; VYNDAMAX | Orphan Designation | - |
| penoscrotal transposition | VYNDAQEL; VYNDAMAX | Orphan Designation | - |
| symptomatic form of Coffin-Lowry syndrome in female carriers | VYNDAQEL; VYNDAMAX | Orphan Designation | - |
| symptomatic form of X-linked centronuclear myopathy in female carriers | VYNDAQEL; VYNDAMAX | Orphan Designation | - |
| symptomatic form of fragile X syndrome in female carrier | VYNDAQEL; VYNDAMAX | Orphan Designation | - |
| symptomatic form of hemophilia A in female carriers | VYNDAQEL; VYNDAMAX | Orphan Designation | - |