7
Orphan Designations
3
FDA Approvals
15
Rare Diseases
0
News Articles
| Disease | Drug(s) | Designation | Approved |
|---|---|---|---|
| Charcot-Marie-Tooth disease axonal type 2S | synthetic 2'-O-methoxyethyl phosphorothioate oligoribonucleotide sodium salt consisting of 19 nucleotide residues with the sequence 5'-MeCMeUGMeUGGAAGMeUGAGGGMeCMeCAG-3' | Orphan Designation | - |
| Charcot-Marie-Tooth disease, axonal, type 2EE | synthetic 2'-O-methoxyethyl phosphorothioate oligoribonucleotide sodium salt consisting of 19 nucleotide residues with the sequence 5'-MeCMeUGMeUGGAAGMeUGAGGGMeCMeCAG-3' | Orphan Designation | - |
| Charcot-Marie-Tooth disease, axonal, type 2FF | synthetic 2'-O-methoxyethyl phosphorothioate oligoribonucleotide sodium salt consisting of 19 nucleotide residues with the sequence 5'-MeCMeUGMeUGGAAGMeUGAGGGMeCMeCAG-3' | Orphan Designation | - |
| FOXG1 disorder | Hetlioz | Orphan Designation | - |
| acquired polycythemia vera | synthetic 19-mer peptide nucleic acid oligomer consisting of two guanine bases possessing a 5-aminopentyl modification, four adenine bases with a 6-aminohexyl moiety, and a fluorenylethoxycarbonyl (Fethoc) group at the 5' terminal end of the sequencesynthetic 2-O-methoxyethyl phosphorothioate oligoribonucleotide sodium salt consisting of 19 nucleotide residues with the sequence 5'-AMeUAGMeUMeUMeCAMeUAAMeUMeCMeUGGAGA-3' | Orphan Designation | - |
| cerebellar dysfunction with variable cognitive and behavioral abnormalities | HETLIOZ | Orphan Designation | - |
| cholera | (+-)-9-(5-bromofuran-2-yl)-12,14-dimethyl-13,15-dioxo-17-phenyl-8-oxa-1,12,14-triazatetracyclo[8.7.0.02,7.011,16]heptadeca-2(7),3,5,10,16-pentaene-4-carboxylic acid | Orphan Designation | - |
| developmental and epileptic encephalopathy, 24 | HETLIOZ | Orphan Designation | - |
| generalized pustular psoriasis | imsidolimab | Orphan Designation | - |
| intellectual disability, X-linked, syndromic 33 | Hetlioz | Orphan Designation | - |
| intellectual disability, autosomal dominant 24 | HETLIOZ | Orphan Designation | - |
| intellectual disability, autosomal dominant 29 | Hetlioz | Orphan Designation | - |
| intellectual disability, autosomal recessive 18 | Hetlioz | Orphan Designation | - |
| non-24-hour sleep-wake syndrome | HETLIOZ | Orphan Designation | - |
| non-Zellweger spectrum disorder | HETLIOZ | Orphan Designation | - |