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ARO-DUX4 (SRP-1001) dose-escalation study in adults with facioscapulohumeral muscular dystrophy type 1
NL-OMON60299Phase 2Active, not recruiting
- Sponsor
- Sarepta Therapeutics
- Start
- Jun 2025
- 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
- 1
01
Overview
Phase 2, open-label, single-arm trial of ARO-DUX4 in facioscapulohumeral muscular dystrophy (FSHD), sponsored by Sarepta Therapeutics. Primary endpoint: Incidence, frequency, and severity of treatment-emergent adverse events. Started Jun 2025; status: active, not recruiting.
02
Drugs
ARO-DUX4
03
Conditions
facioscapulohumeral muscular dystrophy (FSHD)
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