A pharmaceutical company engaged in the development and commercialization of treatments for rare diseases, with a focus on conditions such as renal tubular acidosis and DYRK1A-related intellectual disability syndrome.
1
Orphan Designations
11
FDA Approvals
4
Rare Diseases
0
News Articles
| Disease | Drug(s) | Designation | Approved |
|---|---|---|---|
| DYRK1A-related intellectual disability syndrome | Antipyrine Test | Orphan Designation | - |
| anosmia for isobutyric acid | Antipyrine Test | Orphan Designation | - |
| neuroacanthocytosis | Antipyrine Test | Orphan Designation | - |
| renal tubular acidosis | POTASSIUM CITRATE | - | FDA Approved |