| Englischer Name | Forschungscode | Forschungsphase | Firma | Indikationen | Klinische Studie |
|---|---|---|---|---|---|
| Delpacibart eteddesiran | AOC-1001; AOC 1001 | Phase 3 Clinical | Avidity Biosciences Inc | Myotonic Disorders; Genetic Diseases, Inborn; Musculoskeletal Diseases; Muscular Disorders, Atrophic; Myotonic Dystrophy; Muscular Atrophy; Heredodegenerative Disorders, Nervous System; Muscular Diseases; Neuromuscular Diseases; Nervous System Diseases; Muscular Dystrophies; Neurodegenerative Diseases; Myotonia | Details |
| Dyne-101 | Dyne101 | Phase 2 Clinical | Dyne Therapeutics Inc | Myotonic Dystrophy | Details |
| ARO-DM1 | ARO-DM1 | Phase 2 Clinical | Arrowhead Pharmaceuticals Inc | Myotonic Dystrophy | Details |