1
Orphan Designations
0
FDA Approvals
5
Rare Diseases
0
News Articles
| Disease | Drug(s) | Designation | Approved |
|---|---|---|---|
| frontotemporal dementia and/or amyotrophic lateral sclerosis 7 | Krintafel | Orphan Designation | - |
| malaria | Krintafel | Orphan Designation | - |
| methylmalonic aciduria and homocystinuria type cblF | Krintafel | Orphan Designation | - |
| neurodegeneration with brain iron accumulation 5 | Krintafel | Orphan Designation | - |
| primary release disorder of platelets | Krintafel | Orphan Designation | - |