3
Orphan Designations
0
FDA Approvals
9
Rare Diseases
0
News Articles
| Disease | Drug(s) | Designation | Approved |
|---|---|---|---|
| H syndrome | Adeno-associated viral vector encoding the human frataxin (FXN) gene, AAVrh.10hFXN | Orphan Designation | - |
| Naxos disease | adeno-associated virus serotype rh.10 encoding for the human PKP2 gene (AAVrh.10hPKP2) | Orphan Designation | - |
| THOC6-related developmental delay-microcephaly-facial dysmorphism syndrome | Adeno-associated viral vector encoding the human frataxin (FXN) gene, AAVrh.10hFXN | Orphan Designation | - |
| arrhythmogenic cardiomyopathy with variable ectodermal abnormalities | adeno-associated virus serotype rh.10 encoding for the human PKP2 gene (AAVrh.10hPKP2) | Orphan Designation | - |
| ceroid lipofuscinosis, neuronal, 4 (Kufs type) | Adeno-associated viral vector encoding the human ceroid lipofuscinosis neuronal type 2 (CLN2) gene | Orphan Designation | - |
| ceroid lipofuscinosis, neuronal, 6B (Kufs type) | Adeno-associated viral vector encoding the human ceroid lipofuscinosis neuronal type 2 (CLN2) gene | Orphan Designation | - |
| heterotaxy, visceral, 1, X-linked | Adeno-associated viral vector encoding the human frataxin (FXN) gene, AAVrh.10hFXN | Orphan Designation | - |
| myofibrillar myopathy 1 | adeno-associated virus serotype rh.10 encoding for the human PKP2 gene (AAVrh.10hPKP2) | Orphan Designation | - |
| neuronal ceroid lipofuscinosis 2 | Adeno-associated viral vector encoding the human ceroid lipofuscinosis neuronal type 2 (CLN2) gene | Orphan Designation | - |