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GUARDIAN long-term vamorolone study in boys with Duchenne muscular dystrophy
2024-512828-12-00Phase 4Authorised
- Sponsor
- Santhera Pharmaceuticals
- Start
- Dec 2024
- 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
- 9
01
Overview
Phase 4, open-label, single-arm trial of viltolarsen in Duchenne muscular dystrophy (DMD), sponsored by Santhera Pharmaceuticals. Primary endpoint: Number of vertebral fractures per 1000 person-years. Planned start Dec 2024; status: authorised.
02
Drugs
viltolarsen
03
Conditions
Duchenne muscular dystrophy (DMD)
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