Drug Type ASO |
Synonyms + [1] |
Target |
Action modulators, stimulants |
Mechanism DMD exon 44 modulators(Dystrophin exon 44 modulators), Dystrophin expression stimulants, RNA interference |
Therapeutic Areas |
Inactive Indication- |
Originator Organization |
Active Organization |
Inactive Organization- |
Drug Highest PhasePhase 2 |
First Approval Date- |
RegulationRare Pediatric Disease (United States), Orphan Drug (European Union), Breakthrough Therapy (United States) |
Indication | Highest Phase | Country/Location | Organization | Date |
---|---|---|---|---|
Muscular Dystrophy, Duchenne | Phase 2 | Japan | 23 Jun 2021 | |
Exon 44 Skipping Mutation Duchenne Muscular Dystrophy | Phase 2 | - | - |
Phase 1/2 | Muscular Dystrophy, Duchenne peptidyl arginine deiminase 2 (PADI2) | titin (TTN) | myomesin 2 (MYOM2) | 6 | nhfipmopuw(ubuxccmbbm) = jabeyvhwoa ybbqfdchey (tgdwcbddyu ) | Positive | 01 Jan 2025 | ||
nhfipmopuw(ubuxccmbbm) = voimxjwudy ybbqfdchey (tgdwcbddyu ) | |||||||
Phase 2 | Muscular Dystrophy, Duchenne dystrophin gene mutation amenable to exon 44 skipping | 20 | coevnoisct(sraehpivlb) = grgpzyibpk cojhrvlnmp (ivwjzukuyq ) | Positive | 03 Mar 2024 |