10
Orphan Designations
1
FDA Approvals
18
Rare Diseases
0
News Articles
| Disease | Drug(s) | Designation | Approved |
|---|---|---|---|
| GM1 gangliosidosis | N-Acetyl-Leucine | Orphan Designation | - |
| GM2 gangliosidosis | N-acetyl-L-leucineN-acetyl-DL-leucine | Orphan Designation | - |
| Joubert syndrome with renal defect | N-Acetyl-Leucine | Orphan Designation | - |
| Niemann-Pick disease, type C1 | AqneursaUrsodeoxycholic acidN-acetyl-DL-leucine | Orphan Designation | - |
| ataxia telangiectasia | N-Acetyl-Leucine | Orphan Designation | - |
| autosomal dominant cerebellar ataxia | N-acetyl-DL-leucine | Orphan Designation | - |
| multiple system atrophy | N-Acetyl-Leucine | Orphan Designation | - |
| multiple system atrophy, cerebellar type | N-Acetyl-Leucine | Orphan Designation | - |
| multiple system atrophy, parkinsonian type | N-Acetyl-Leucine | Orphan Designation | - |
| neurological pain disorder | Aqneursa | Orphan Designation | - |
| pediatric acute-onset neuropsychiatric syndrome | Aqneursa | Orphan Designation | - |
| pediatric hepatocellular carcinoma | Aqneursa | Orphan Designation | - |
| pediatric ovarian dysgerminoma | Aqneursa | Orphan Designation | - |
| pediatric ovarian germ cell tumor | Aqneursa | Orphan Designation | - |
| pediatric-onset Graves disease | Aqneursa | Orphan Designation | - |
| pellagra-like syndrome | Aqneursa | Orphan Designation | - |
| spinocerebellar ataxia 43 | N-acetyl-DL-leucine | Orphan Designation | - |
| spinocerebellar ataxia 7 | N-acetyl-DL-leucine | Orphan Designation | - |