2
Orphan Designations
0
FDA Approvals
6
Rare Diseases
0
News Articles
| Disease | Drug(s) | Designation | Approved |
|---|---|---|---|
| Cornelia de Lange syndrome 1 | ecopipam hydrochloride | Orphan Designation | - |
| intellectual disability-severe speech delay-mild dysmorphism syndrome | ecopipam hydrochloride | Orphan Designation | - |
| microcephaly 9, primary, autosomal recessive | ecopipam hydrochloride | Orphan Designation | - |
| pediatric hepatocellular carcinoma | ecopipam hydrochloride | Orphan Designation | - |
| pediatric ovarian dysgerminoma | ecopipam hydrochloride | Orphan Designation | - |
| pediatric-onset Graves disease | ecopipam hydrochloride | Orphan Designation | - |