A Study of Long-term Safety and Efficacy of VX-670 in Participants With Myotonic Dystrophy Type I

Study Summary

The purpose of the study is to evaluate the long-term safety and tolerability, efficacy and pharmacokinetics of VX-670 in participants with Myotonic Dystrophy Type I (DM1).

Conditions

Myotonic Dystrophy Type 1 (DM1)

Interventions / Drugs

VX-670

View on ClinicalTrials.gov ↗

Sponsor

VRTX

Vertex Pharmaceuticals

Gene Therapy

C55/100

Study Details

Enrollment44 participants

Primary Completion—

Lead SponsorVertex Pharmaceuticals Incorporated

Data Retrieved2026-04-11 02:55:58.035248+00:00

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