10
Orphan Designations
2
FDA Approvals
24
Rare Diseases
0
News Articles
| Disease | Drug(s) | Designation | Approved |
|---|---|---|---|
| Angelman syndrome | Chimeric locked nucleic acid and ribonucleic-deoxyribonucleic antisense oligonucleotide specific for the human UBE3A-antisense transcript | Orphan Designation | - |
| Dorfman-Chanarin disease | Dojolvi | Orphan Designation | - |
| Wilson disease | Adeno-associated viral vector serotype 9 encoding human ATP7B | Orphan Designation | - |
| adult-onset non-insulinoma persistent hyperinsulinemic hypoglycemia | Dojolvi | Orphan Designation | - |
| cystinosis | triheptanoin | Orphan Designation | - |
| disorder of fatty acid oxidation and ketogenesis | Dojolvi | Orphan Designation | - |
| fatty acid hydroxylase-associated neurodegeneration | Dojolvi | Orphan Designation | - |
| glucose transport disorder | triheptanoin | Orphan Designation | - |
| glycogen storage disease III | mRNA encoding the human glycogen debranching enzyme | Orphan Designation | - |
| glycogen storage disease IX | recombinant adeno-associated virus serotype 8 vector encoding human glucose-6-phosphatase-alpha (G6Pase or G6PC) | Orphan Designation | - |
| glycogen storage disease IXc | recombinant adeno-associated virus serotype 8 vector encoding human glucose-6-phosphatase-alpha (G6Pase or G6PC) | Orphan Designation | - |
| glycogen storage disease Ib | recombinant adeno-associated virus serotype 8 vector encoding human glucose-6-phosphatase-alpha (G6Pase or G6PC)triheptanoin | Orphan Designation | - |
| hereditary inclusion body myopathy-joint contractures-ophthalmoplegia syndrome | aceneuramic acid | Orphan Designation | - |
| inherited fatty acid metabolism disorder | Dojolvi | Orphan Designation | - |
| mucopolysaccharidosis | Mepsevii | Orphan Designation | - |
| mucopolysaccharidosis type 6 | Mepsevii | Orphan Designation | - |
| mucopolysaccharidosis type 6, rapidly progressing | Mepsevii | Orphan Designation | - |
| mucopolysaccharidosis type 6, slowly progressing | Mepsevii | Orphan Designation | - |
| mucopolysaccharidosis type 7 | Mepsevii | Orphan Designation | - |
| osteogenesis imperfecta | human monoclonal antibody targeting human sclerostin | Orphan Designation | - |
| pediatric hepatocellular carcinoma | Dojolvi | Orphan Designation | - |
| pediatric ovarian dysgerminoma | Dojolvi | Orphan Designation | - |
| pediatric-onset Graves disease | Dojolvi | Orphan Designation | - |
| primary adult heart tumor | Dojolvi | Orphan Designation | - |