2
Orphan Designations
0
FDA Approvals
1
Rare Diseases
0
News Articles
| Disease | Drug(s) | Designation | Approved |
|---|---|---|---|
| Riley-Day syndrome | 2-[(2S)-2-aminopropyl]-3,5-dichloro-N-(2-thienylmethyl)thieno[3,2-b]pyridin-7-amineRecombinant adeno-associated virus serotype 2 containing the human ELP1 gene, driven by the U1a promoter | Orphan Designation | - |