2
Orphan Designations
0
FDA Approvals
7
Rare Diseases
0
News Articles
| Disease | Drug(s) | Designation | Approved |
|---|---|---|---|
| Fanconi renotubular syndrome 2 | extracellular domain of Ectonucleotide Pyrophosphatase/Phosphodiesterase 1 fused to the Fc region of human IgG1 | Orphan Designation | - |
| Tangier disease | Recombinant human ectonucleotide pyrophosphatase/phosphodiesterase 1-Fc (rhENPP1-Fc) enzyme | Orphan Designation | - |
| adenosine kinase deficiency | Recombinant human ectonucleotide pyrophosphatase/phosphodiesterase 1-Fc (rhENPP1-Fc) enzyme | Orphan Designation | - |
| arterial calcification, generalized, of infancy, 2 | extracellular domain of Ectonucleotide Pyrophosphatase/Phosphodiesterase 1 fused to the Fc region of human IgG1 | Orphan Designation | - |
| hypophosphatemic rickets, autosomal recessive, 2 | extracellular domain of Ectonucleotide Pyrophosphatase/Phosphodiesterase 1 fused to the Fc region of human IgG1 | Orphan Designation | - |
| pyruvate kinase hyperactivity | Recombinant human ectonucleotide pyrophosphatase/phosphodiesterase 1-Fc (rhENPP1-Fc) enzyme | Orphan Designation | - |
| vitamin D-dependent rickets, type 2A | extracellular domain of Ectonucleotide Pyrophosphatase/Phosphodiesterase 1 fused to the Fc region of human IgG1 | Orphan Designation | - |