2
Orphan Designations
0
FDA Approvals
5
Rare Diseases
0
News Articles
| Disease | Drug(s) | Designation | Approved |
|---|---|---|---|
| brain-lung-thyroid syndrome | human cystic fibrosis transmembrane conductance regulator (hCFTR) messenger RNA (mRNA) complexed with branched polyethyleneimine (bPEI) | Orphan Designation | - |
| lymphedema-distichiasis syndrome | human cystic fibrosis transmembrane conductance regulator (hCFTR) messenger RNA (mRNA) complexed with branched polyethyleneimine (bPEI) | Orphan Designation | - |
| ornithine aminotransferase deficiency | Nanoparticle suspension containing biosynthetic codon-optimized human ornithine transcarbamylase messenger RNA | Orphan Designation | - |
| ornithine carbamoyltransferase deficiency | Nanoparticle suspension containing biosynthetic codon-optimized human ornithine transcarbamylase messenger RNA | Orphan Designation | - |
| ornithine translocase deficiency | Nanoparticle suspension containing biosynthetic codon-optimized human ornithine transcarbamylase messenger RNA | Orphan Designation | - |