3
Orphan Designations
0
FDA Approvals
7
Rare Diseases
0
News Articles
| Disease | Drug(s) | Designation | Approved |
|---|---|---|---|
| GM2 gangliosidosis | adeno-associated viral vector serotype 9 (AAV9) carrying both HEXA and HEXB | Orphan Designation | - |
| ceroid lipofuscinosis, neuronal, 6B (Kufs type) | a self-complementary adeno-associated virus serotype 9 expressing human codon-optimized CLN1 under control of CBh | Orphan Designation | - |
| malignant carotid body paraganglioma | recombinant serotype 9 adeno-associated virus encoding a codon-optimized human MECP2 gene | Orphan Designation | - |
| neuronal ceroid lipofuscinosis 1 | a self-complementary adeno-associated virus serotype 9 expressing human codon-optimized CLN1 under control of CBh | Orphan Designation | - |
| neuronal ceroid lipofuscinosis 2 | a self-complementary adeno-associated virus serotype 9 expressing human codon-optimized CLN1 under control of CBh | Orphan Designation | - |
| post-treatment Lyme disease syndrome | recombinant serotype 9 adeno-associated virus encoding a codon-optimized human MECP2 gene | Orphan Designation | - |
| treatment-refractory schizophrenia | recombinant serotype 9 adeno-associated virus encoding a codon-optimized human MECP2 gene | Orphan Designation | - |