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DYNE-101 multiple ascending dose study in myotonic dystrophy type 1
2023-510353-42-00Phase 2Authorised
- Sponsor
- Dyne Therapeutics
- Start
- Feb 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
- 8
01
Overview
Phase 2, randomised trial in myotonic dystrophy type 1 (DM1) comparing DYNE-101 with Placebo (2 arms). Primary endpoints: Safety and tolerability and Change from baseline in myotonia by vHOT at Week 25. Planned start Feb 2022; status: authorised.
02
Drugs
zeleciment basivarsen
03
Conditions
myotonic dystrophy type 1 (DM1)
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