Drug Type Gene editing |
Synonyms- |
Target |
Mechanism telethonin stimulants(titin-cap stimulants) |
Therapeutic Areas |
Active Indication |
Inactive Indication- |
Originator Organization Cure Rare Disease, Inc.Startup |
Active Organization Cure Rare Disease, Inc.Startup |
Inactive Organization- |
Drug Highest PhasePreclinical |
First Approval Date- |
Regulation- |
Indication | Highest Phase | Country/Location | Organization | Date |
---|---|---|---|---|
Muscular Dystrophy, Limb-Girdle, Type 2G | Preclinical | US | Cure Rare Disease, Inc.Startup | 11 Apr 2024 |