2
Orphan Designations
0
FDA Approvals
4
Rare Diseases
0
News Articles
| Disease | Drug(s) | Designation | Approved |
|---|---|---|---|
| Wolf-Hirschhorn syndrome | L-cysteine | Orphan Designation | - |
| double nail for fifth toe | L-cysteine | Orphan Designation | - |
| malignant glioma | replication-competent oncolytic herpes simplex virus (oHSV), engineered by removing the two endogenous copies of infected cell protein 34.5 (ICP34.5) and re-inserting one copy of the ICP34.5 gene under transcriptional control mediated by the promoter-enhancer elements of nestin | Orphan Designation | - |
| neurodegeneration with brain iron accumulation 2A | L-cysteine | Orphan Designation | - |