4
Orphan Designations
0
FDA Approvals
13
Rare Diseases
0
News Articles
| Disease | Drug(s) | Designation | Approved |
|---|---|---|---|
| Bicervical bicornuate uterus with patent cervix and vagina | Rilutek | Orphan Designation | - |
| Huntington disease | Riluzole | Orphan Designation | - |
| amyotrophic lateral sclerosis | Rilutek | Orphan Designation | - |
| carbon monoxide-induced delayed encephalopathy | Rilutek | Orphan Designation | - |
| mitochondrial DNA deletion syndrome with progressive myopathy | Rilutek | Orphan Designation | - |
| multiple congenital anomalies-hypotonia-seizures syndrome 2 | Rilutek | Orphan Designation | - |
| myoglobinuria, acute recurrent, autosomal recessive | Spiramycin | Orphan Designation | - |
| parasitic Ichthyosporea infectious disease | Spiramycin | Orphan Designation | - |
| parasitic ectoparasitic infectious disease | Spiramycin | Orphan Designation | - |
| parasitic eye infection | Spiramycin | Orphan Designation | - |
| parasitic intestinal disorder | Spiramycin | Orphan Designation | - |
| spinal muscular atrophy with congenital bone fractures 1 | Rilutek | Orphan Designation | - |
| symptomatic form of hemophilia A in female carriers | Spiramycin | Orphan Designation | - |