4
Orphan Designations
0
FDA Approvals
10
Rare Diseases
1
News Articles
| Disease | Drug(s) | Designation | Approved |
|---|---|---|---|
| Leber congenital amaurosis | adenovirus associated viral vector serotype 5 containing the human RPE65 gene | Orphan Designation | - |
| achromatopsia | adenovirus associated viral vector serotype 8 containing the human Cyclic Nucleotide Gated Channel Alpha 3 subunit (CNGA3) geneadenovirus associated viral vector serotype 8 containing the human CNGB3 gene | Orphan Designation | - |
| achromatopsia 2 | adenovirus associated viral vector serotype 8 containing the human Cyclic Nucleotide Gated Channel Alpha 3 subunit (CNGA3) geneadenovirus associated viral vector serotype 8 containing the human CNGB3 gene | Orphan Designation | - |
| achromatopsia 4 | adenovirus associated viral vector serotype 8 containing the human Cyclic Nucleotide Gated Channel Alpha 3 subunit (CNGA3) geneadenovirus associated viral vector serotype 8 containing the human CNGB3 gene | Orphan Designation | - |
| acute intermittent porphyria | replication-deficient recombinant serotype 2 adeno-associated viral vector containing hAQP1 cDNA | Orphan Designation | - |
| developmental and epileptic encephalopathy, 41 | replication-deficient recombinant serotype 2 adeno-associated viral vector containing hAQP1 cDNA | Orphan Designation | - |
| hereditary coproporphyria | replication-deficient recombinant serotype 2 adeno-associated viral vector containing hAQP1 cDNA | Orphan Designation | - |
| oligodendroglioma, IDH-mutant and 1p/19q-codeleted, grade 3 | replication-deficient recombinant serotype 2 adeno-associated viral vector containing hAQP1 cDNA | Orphan Designation | - |
| tumor grade 3 or 4, general grading system | replication-deficient recombinant serotype 2 adeno-associated viral vector containing hAQP1 cDNA | Orphan Designation | - |
| tumor grade 3, general grading system | replication-deficient recombinant serotype 2 adeno-associated viral vector containing hAQP1 cDNA | Orphan Designation | - |