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ARO-DM1 phase 1/2a dose-escalation study in adults with type 1 myotonic dystrophy
2024-513579-42-00Phase 2Authorised
- Sponsor
- Sarepta Therapeutics
- Start
- Apr 2026
- Modality
- 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 2, open-label, single-arm trial of SRP-1003 in myotonic dystrophy type 1 (DM1), sponsored by Sarepta Therapeutics. Primary endpoint: Incidence, frequency, and severity of treatment-emergent adverse events and treatment-related adverse events. Planned start Apr 2026; status: authorised.
02
Drugs
SRP-1003
03
Conditions
myotonic dystrophy type 1 (DM1)
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