3
Orphan Designations
0
FDA Approvals
5
Rare Diseases
2
News Articles
| Disease | Drug(s) | Designation | Approved |
|---|---|---|---|
| Crigler-Najjar syndrome | modified mRNA encoding UGT1A1 protein, formulated in lipid nanoparticles | Orphan Designation | - |
| isolated cryptophthalmia | messanger ribonucleic acid (mRNA)-based therapeutic agent encoding human methylmalonyl-coenzyme A mutase | Orphan Designation | - |
| isolated methylmalonic aciduria cblD type | messanger ribonucleic acid (mRNA)-based therapeutic agent encoding human methylmalonyl-coenzyme A mutase | Orphan Designation | - |
| isolated tracheo-esophageal fistula | messanger ribonucleic acid (mRNA)-based therapeutic agent encoding human methylmalonyl-coenzyme A mutase | Orphan Designation | - |
| propionic acidemia | messenger ribonucleic acid-based therapeutic agent encoding human propionyl-CoA carboxylase alpha and beta subunits | Orphan Designation | - |