7
Orphan Designations
2
FDA Approvals
12
Rare Diseases
0
News Articles
| Disease | Drug(s) | Designation | Approved |
|---|---|---|---|
| Becker muscular dystrophy | Agamree | Orphan Designation | - |
| Duchenne muscular dystrophy | Agamree | Orphan Designation | - |
| Lambert syndrome | Firdapse | Orphan Designation | - |
| Lambert-Eaton myasthenic syndrome | Firdapseamifampridine | Orphan Designation | - |
| Schwartz-Cohen-addad-Lambert syndrome | Firdapse | Orphan Designation | - |
| congenital myasthenic syndrome | 3,4-diaminopyridineamifampridine phosphate | Orphan Designation | - |
| congenital myasthenic syndrome 11 | 3,4-diaminopyridine | Orphan Designation | - |
| congenital myasthenic syndrome 5 | 3,4-diaminopyridine | Orphan Designation | - |
| ichthyosis histrix, Lambert type | Firdapse | Orphan Designation | - |
| infantile spasms | (1S,3S)-3-amino-4-(difluoromethylene)cyclopentanecarboxylic acid hydrochloride, (1S,3S)-3-amino-4-difluoromethylenyl-1-cyclopentanoic acid hydrochloride | Orphan Designation | - |
| myasthenia gravis | amifampridine phosphate | Orphan Designation | - |
| symptomatic form of muscular dystrophy of Duchenne and Becker in female carriers | Agamree | Orphan Designation | - |