8
Orphan Designations
0
FDA Approvals
17
Rare Diseases
0
News Articles
| Disease | Drug(s) | Designation | Approved |
|---|---|---|---|
| 3-hydroxy-3-methylglutaric aciduria | Oxandrolone | Orphan Designation | - |
| Alexander disease | Testosterone sublingual | Orphan Designation | - |
| Faundes-Banka syndrome | Testosterone sublingual | Orphan Designation | - |
| Meier-Gorlin syndrome 6 | Testosterone sublingual | Orphan Designation | - |
| Noonan syndrome | Ethinyl Estradiol, USP | Orphan Designation | - |
| Turner syndrome | Ethinyl Estradiol, USP | Orphan Designation | - |
| chronic asthma | Oxandrolone | Orphan Designation | - |
| constitutional delay of growth and puberty | Testosterone sublingualOxandrolone | Orphan Designation | - |
| diencephalic syndrome | Somatropin | Orphan Designation | - |
| ischemia reperfusion injury | Superoxide dismutase (recombinant human) | Orphan Designation | - |
| malocclusion and short stature | Oxandrolone | Orphan Designation | - |
| mitochondrial DNA depletion syndrome 6 (hepatocerebral type) | Oxandrolone | Orphan Designation | - |
| mitochondrial neurogastrointestinal encephalomyopathy | Somatropin | Orphan Designation | - |
| nail-patella syndrome | Ethinyl Estradiol, USP | Orphan Designation | - |
| short stature, Brussels type | Oxandrolone | Orphan Designation | - |
| skeletal dysplasia-epilepsy-short stature syndrome | Oxandrolone | Orphan Designation | - |
| syndromic X-linked intellectual disability 34 | Testosterone sublingual | Orphan Designation | - |