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Phase II open-label multiple-dose study of Agamree in boys with Duchenne muscular dystrophy
2025-000201-16Phase 2
- Sponsor
- Santhera Pharmaceuticals
- Start
- May 2025
- Modality
- Small Interfering RNA (siRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, MicroRNA (miRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Antisense Oligonucleotides (ASOs) → Long RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics
- Countries
- 1
01
Overview
Phase 2, open-label trial of viltolarsen in Duchenne muscular dystrophy (DMD) (2 arms), sponsored by Santhera Pharmaceuticals. Primary endpoint: Safety endpoints.
02
Drugs
viltolarsen
03
Conditions
Duchenne muscular dystrophy (DMD)
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