2
Orphan Designations
2
FDA Approvals
11
Rare Diseases
0
News Articles
| Disease | Drug(s) | Designation | Approved |
|---|---|---|---|
| Bicervical bicornuate uterus with patent cervix and vagina | Purixan | Orphan Designation | - |
| Down syndrome | Purixan | Orphan Designation | - |
| RNU5B-1 related neurodevelopmental disorder with seizures and joint laxity | Xromi | Orphan Designation | - |
| acute lymphoblastic leukemia | Purixan | Orphan Designation | - |
| carbon monoxide-induced delayed encephalopathy | Purixan | Orphan Designation | - |
| multiple congenital anomalies-hypotonia-seizures syndrome 2 | Purixan | Orphan Designation | - |
| natal teeth-intestinal pseudoobstruction-patent ductus syndrome | Purixan | Orphan Designation | - |
| sickle cell disease | Xromi | Orphan Designation | - |
| spastic paraplegia 93, autosomal recessive | Xromi | Orphan Designation | - |
| spinocerebellar ataxia, autosomal recessive 33 | Xromi | Orphan Designation | - |
| syndromic X-linked intellectual disability 5 | Xromi | Orphan Designation | - |