15
Orphan Designations
0
FDA Approvals
15
Rare Diseases
0
News Articles
| Disease | Drug(s) | Designation | Approved |
|---|---|---|---|
| Becker muscular dystrophy | nandrolone | Orphan Designation | - |
| Duchenne muscular dystrophy | Prednisonetestosteroneinsulin-like growth factor-1nandrolone | Orphan Designation | - |
| Erb palsy | nandrolone | Orphan Designation | - |
| Kennedy disease | Insulin-like Growth Factor-1 | Orphan Designation | - |
| Phelan-McDermid syndrome | insulin-like growth factor-1(IGF-1) | Orphan Designation | - |
| amyotrophic lateral sclerosis | pegylated insulin-like growth factor I | Orphan Designation | - |
| chronic inflammatory demyelinating polyradiculoneuropathy | prednisone | Orphan Designation | - |
| congenital muscular dystrophy | Insulin-like Growth Factor-1 | Orphan Designation | - |
| facioscapulohumeral muscular dystrophy | nandrolone | Orphan Designation | - |
| limb-girdle muscular dystrophy | Prednisonenandrolone | Orphan Designation | - |
| myasthenia gravis | prednisone | Orphan Designation | - |
| myotonic dystrophy type 1 | mecasermin | Orphan Designation | - |
| spinal muscular atrophy, type II | pegylated insulin-like growth factor 1 (PEG-IGF1) | Orphan Designation | - |
| spinal muscular atrophy, type III | pegylated insulin-like growth factor 1 (PEG-IGF1) | Orphan Designation | - |
| spinal muscular atrophy, type IV | pegylated insulin-like growth factor 1 (PEG-IGF1) | Orphan Designation | - |