2
Orphan Designations
1
FDA Approvals
2
Rare Diseases
1
News Articles
| Disease | Drug(s) | Designation | Approved |
|---|---|---|---|
| amyotrophic lateral sclerosis | autologous adipose derived mesenchymal stromal cells | Orphan Designation | - |
| propionic acidemia | Adeno-Associated Viral Vector Expressing Codon-optimized Human PCCA cDNA | Orphan Designation | - |