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Phase 1/2a study of ATX-01 in adults with myotonic dystrophy type 1
NL-OMON60957Phase 1Recruiting
- Sponsor
- ARTHEx Biotech
- Start
- Sep 2025
- Modality
- Gene Therapies, Antisense Oligonucleotides (ASOs) → Long RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Single-strand DNA → Antisense DNA → DNA Therapeutics → Nucleic Acid Therapeutics
- Countries
- 1
01
Overview
Phase 1, open-label trial of ATX-01 in myotonic dystrophy type 1 (DM1) (2 arms), sponsored by ARTHEx Biotech. Primary endpoints: Incidence of Serious Adverse Events, Incidence of Adverse Events of Special Interest and 2 more. Started Sep 2025; status: recruiting.
02
Drugs
ATX-01
03
Conditions
myotonic dystrophy type 1 (DM1)
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