1
Orphan Designations
0
FDA Approvals
3
Rare Diseases
0
News Articles
| Disease | Drug(s) | Designation | Approved |
|---|---|---|---|
| intellectual disability-spasticity-ectrodactyly syndrome | L-threonine | Orphan Designation | - |
| microcephaly-brain defect-spasticity-hypernatremia syndrome | L-threonine | Orphan Designation | - |
| spasticity-ataxia-gait anomalies syndrome | L-threonine | Orphan Designation | - |