A biopharmaceutical company focused on developing treatments for rare diseases, with 21 FDA-approved drugs and involvement in conditions such as adult-onset proximal spinal muscular atrophy and chronic myelogenous leukemia.
1
Orphan Designations
21
FDA Approvals
6
Rare Diseases
0
News Articles
| Disease | Drug(s) | Designation | Approved |
|---|---|---|---|
| adult neuronal ceroid lipofuscinosis | Danziten | Orphan Designation | - |
| adult pleomorphic rhabdomyosarcoma | Danziten | Orphan Designation | - |
| adult-onset non-insulinoma persistent hyperinsulinemic hypoglycemia | Danziten | Orphan Designation | - |
| adult-onset proximal spinal muscular atrophy, autosomal dominant | Danziten | Orphan Designation | - |
| chronic myelogenous leukemia, BCR-ABL1 positive | Danziten | Orphan Designation | - |
| primary adult heart tumor | Danziten | Orphan Designation | - |