2
Orphan Designations
0
FDA Approvals
4
Rare Diseases
0
News Articles
| Disease | Drug(s) | Designation | Approved |
|---|---|---|---|
| beta-thalassemia HBB/LCRB | 6-[(3S,4S)-4-methyl-1-(pyrimidin-2-ylmethyl)pyrrolidin-3-yl]-3- tetrahydropyran-4-yl-7H-imidazo[1,5-a]pyrazin-8-one | Orphan Designation | - |
| sickle cell disease | 6-[(3S,4S-4-methyl-1-(pyrimidin-2-ylmethyl)pyrrolidin-3-yl]-3-tetrahydropyran-4-yl-7H-imadazo[1,5-a]pyrazin-8-one | Orphan Designation | - |
| sickle cell-hemoglobin c disease syndrome | 6-[(3S,4S-4-methyl-1-(pyrimidin-2-ylmethyl)pyrrolidin-3-yl]-3-tetrahydropyran-4-yl-7H-imadazo[1,5-a]pyrazin-8-one | Orphan Designation | - |
| sickle cell-hemoglobin d disease syndrome | 6-[(3S,4S-4-methyl-1-(pyrimidin-2-ylmethyl)pyrrolidin-3-yl]-3-tetrahydropyran-4-yl-7H-imadazo[1,5-a]pyrazin-8-one | Orphan Designation | - |