4
Orphan Designations
0
FDA Approvals
7
Rare Diseases
0
News Articles
| Disease | Drug(s) | Designation | Approved |
|---|---|---|---|
| COG8-congenital disorder of glycosylation | Reviparin sodium | Orphan Designation | - |
| Schimke immuno-osseous dysplasia | Panhematin | Orphan Designation | - |
| amyotrophic lateral sclerosis | Protirelin injection | Orphan Designation | - |
| ataxia-pancytopenia syndrome | Reviparin sodium | Orphan Designation | - |
| chronic thromboembolic pulmonary hypertension | Reviparin sodium | Orphan Designation | - |
| glycogen storage disease II | Panhematin | Orphan Designation | - |
| recurrent metabolic encephalomyopathic crises-rhabdomyolysis-cardiac arrhythmia-intellectual disability syndrome | Reviparin sodium | Orphan Designation | - |