2
Orphan Designations
1
FDA Approvals
11
Rare Diseases
0
News Articles
| Disease | Drug(s) | Designation | Approved |
|---|---|---|---|
| ATTRV30M amyloidosis | Attruby | Orphan Designation | - |
| arrhythmogenic cardiomyopathy with wooly hair and keratoderma | Attruby | Orphan Designation | - |
| familial amyloid neuropathy | Attruby | Orphan Designation | - |
| histiocytoid cardiomyopathy | Attruby | Orphan Designation | - |
| intellectual disability, autosomal dominant 15 | Attruby | Orphan Designation | - |
| malignant carotid body paraganglioma | Encaleret sulfate | Orphan Designation | - |
| microcephaly-cardiomyopathy syndrome | Attruby | Orphan Designation | - |
| myofibrillar myopathy 2 | Attruby | Orphan Designation | - |
| penoscrotal transposition | Attruby | Orphan Designation | - |
| post-treatment Lyme disease syndrome | Encaleret sulfate | Orphan Designation | - |
| treatment-refractory schizophrenia | Encaleret sulfate | Orphan Designation | - |