1
Orphan Designations
0
FDA Approvals
3
Rare Diseases
0
News Articles
| Disease | Drug(s) | Designation | Approved |
|---|---|---|---|
| abetalipoproteinemia | mRNA encoding the cholesterol degrading platform (CDP) fusion protein | Orphan Designation | - |
| homozygous 11P15-p14 deletion syndrome | mRNA encoding the cholesterol degrading platform (CDP) fusion protein | Orphan Designation | - |
| homozygous familial hypercholesterolemia | mRNA encoding the cholesterol degrading platform (CDP) fusion protein | Orphan Designation | - |