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Global Phase 3 Open-Label Extension Study of AOC 1001 in Myotonic Dystrophy Type 1
2025-520766-22-00Phase 3Authorised
- Sponsor
- Avidity Biosciences
- Start
- Sep 2025
- Modality
- Antibody Conjugates → Antibody-Based Therapeutics, Chemically Modified siRNA → Small Interfering RNA (siRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Conjugated siRNA → Small Interfering RNA (siRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics
- Countries
- 10
01
Overview
Phase 3, open-label, single-arm trial of delpacibart etedesiran in myotonic dystrophy type 1 (DM1), sponsored by Avidity Biosciences. Primary endpoint: Incidence of TEAEs. Planned start Sep 2025; status: authorised.
02
Drugs
delpacibart etedesiran
03
Conditions
myotonic dystrophy type 1 (DM1)
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