1
Orphan Designations
0
FDA Approvals
5
Rare Diseases
0
News Articles
| Disease | Drug(s) | Designation | Approved |
|---|---|---|---|
| severe Canavan disease | Alphanate | Orphan Designation | - |
| severe achondroplasia-developmental delay-acanthosis nigricans syndrome | Alphanate | Orphan Designation | - |
| severe intellectual disability-poor language-strabismus-grimacing face-long fingers syndrome | Alphanate | Orphan Designation | - |
| severe neonatal-onset encephalopathy with microcephaly | Alphanate | Orphan Designation | - |
| von Willebrand disease (hereditary or acquired) | Alphanate | Orphan Designation | - |