5
Orphan Designations
0
FDA Approvals
4
Rare Diseases
0
News Articles
| Disease | Drug(s) | Designation | Approved |
|---|---|---|---|
| Duchenne muscular dystrophy | octadecanedioic acid derivative-modified inhibitor of connective tissue growth factor (CTGF) | Orphan Designation | - |
| X-linked dominant hypophosphatemic rickets | DNA, (Cm-Gm-Gm-Gm-G-T-G-T-G-G-G-T-T-C-G-T-C-G-T-T-A-G-C-T-T-G-A-T-T-T-G-G-C-A-G-C-Um-Gm-Cm-Cm-(5'->3')-idT), 5'-ester with (29S)-29-carboxy-8,17,26,31-tetraoxo-10,13,19,22-tetraoxa-7,16,25,30-tetraazaoctatetracontan-48-oic acid | Orphan Designation | - |
| gastric cancer | OA-modified DKK1 aptamer-based PROTAC molecule degrader of DKK1 protein | Orphan Designation | - |
| osteogenesis imperfecta | DNA, (Cm-Gm-Gm-Gm-G-T-G-T-G-G-G-T-T-C-G-T-C-G-T-T-A-G-C-T-T-G-A-T-T-T-G-G-C-A-G-C-Um-Gm-Cm-Cm-(5'->3')-dT), 5'-ester with (29S)-29-(tert-butoxycarbonyl)-1-((hydroxyphosphoryl)oxy)-8,17,26,31-tetraoxo-10,13,19,22-tetraoxa-7,16,25,30-tetraazaoctatetracontan-48-oic acidDNA, (Cm-Gm-Gm-Gm-G-T-G-T-G-G-G-T-T-C-G-T-C-G-T-T-A-G-C-T-T-G-A-T-T-T-G-G-C-A-G-C-Um-Gm-Cm-Cm-(3'¿3')-dT), 5'-ester with [[5-(phosphoonoxy) pentyl] amino] carbonyl-oxy-1, 2-ethanediyl, sodium salt | Orphan Designation | - |