2
Orphan Designations
0
FDA Approvals
2
Rare Diseases
0
News Articles
| Disease | Drug(s) | Designation | Approved |
|---|---|---|---|
| autosomal dominant polycystic kidney disease | fully human IgG1 monoclonal antibody that binds to human pregnancy-associated plasma protein-A (PAPP-A) | Orphan Designation | - |
| leukoencephalopathy with vanishing white matter | Sodium ({(2S)-1,4-bis[2-(4-chloro-3- fluorophenoxy)acetamido]bicyclo[2.2.2]octan-2- yl}oxy)methyl hydrogen phosphate - 2-amino-2- (hydroxymethyl)propane-1,3-diol (1/1/1) | Orphan Designation | - |