1
Orphan Designations
0
FDA Approvals
4
Rare Diseases
0
News Articles
| Disease | Drug(s) | Designation | Approved |
|---|---|---|---|
| abetalipoproteinemia | KYNAMRO | Orphan Designation | - |
| homozygous 11P15-p14 deletion syndrome | KYNAMRO | Orphan Designation | - |
| homozygous familial hypercholesterolemia | KYNAMRO | Orphan Designation | - |
| hypotonia-cystinuria syndrome | KYNAMRO | Orphan Designation | - |