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A Phase 1/2a Dose-Escalating Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of ARO-DM1 in Subjects With Type 1 Myotonic Dystrophy Who Are ≥18 to ≤ 65 Years

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Trial Profile

A Phase 1/2a Dose-Escalating Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of ARO-DM1 in Subjects With Type 1 Myotonic Dystrophy Who Are ≥18 to ≤ 65 Years

Status: Recruiting
Phase of Trial: Phase I/II

Latest Information Update: 13 Oct 2026

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At a glance

  • Drugs SRP 1003 (Primary) ; SRP 1003 (Primary)
  • Indications Myotonic dystrophy
  • Focus Adverse reactions; First in man
  • Sponsors Sarepta Therapeutics

Most Recent Events

  • 11 Aug 2026 Planned End Date changed from 1 Dec 2026 to 31 Dec 2026.
  • 11 Aug 2026 Planned primary completion date changed from 1 Dec 2026 to 31 Dec 2026.
  • 05 Aug 2026 According to a Sarepta Therapeutics media release, readouts from MAD cohort remain on track for 2H 2026.

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