1
Orphan Designations
0
FDA Approvals
6
Rare Diseases
0
News Articles
| Disease | Drug(s) | Designation | Approved |
|---|---|---|---|
| inclusion body myopathy with Paget disease of bone and frontotemporal dementia | Skytrofa | Orphan Designation | - |
| isolated congenital growth hormone deficiency | Skytrofa | Orphan Designation | - |
| isolated growth hormone deficiency type IA | Skytrofa | Orphan Designation | - |
| isolated growth hormone deficiency type IB | Skytrofa | Orphan Designation | - |
| isolated growth hormone deficiency type II | Skytrofa | Orphan Designation | - |
| neuronopathy, distal hereditary motor, autosomal recessive 11, with spasticity | Skytrofa | Orphan Designation | - |