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DYNE-101 in adults with myotonic dystrophy type 1
NL-OMON58695Phase 2Active, not recruiting
- Sponsor
- Dyne Therapeutics
- Start
- May 2022
- Modality
- Antibody Conjugates → Antibody-Based Therapeutics, Conjugated siRNA → Small Interfering RNA (siRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Antisense Oligonucleotides (ASOs) → Long RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics
- Countries
- 1
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Overview
Phase 2, open-label trial of zeleciment basivarsen in myotonic dystrophy type 1 (DM1) (2 arms), sponsored by Dyne Therapeutics. Primary endpoints: Safety and tolerability in MAD cohorts and Change in myotonia by vHOT in dose expansion cohort. Started May 2022; status: active, not recruiting.
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Drugs
zeleciment basivarsen
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Conditions
myotonic dystrophy type 1 (DM1)
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