Gene therapy for duchenne MD: Long-Term safety and strength check

NCT ID NCT06491927

ENROLLING_BY_INVITATION Disease control Sponsor: REGENXBIO Inc. Source: ClinicalTrials.gov ↗

First seen Feb 26, 2026 · Last updated May 02, 2026 · Updated 8 times

Summary

This study follows 66 boys with Duchenne muscular dystrophy who previously received RGX-202 gene therapy in an earlier trial. Researchers will monitor safety and measure how well the therapy helps with standing, walking, and climbing over time. The goal is to see if the treatment remains safe and effective in the long run.

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Contacts and locations

Locations

  • Ann & Robert H. Lurie Children's Hospital of Chicago

    Chicago, Illinois, 60611, United States

  • Arkansas Children's Hospital

    Little Rock, Arkansas, 72202, United States

  • Stanford School of Medicine /Division of Neuromuscular Medicine

    Palo Alto, California, 94304, United States

  • The University of Texas Southwestern Medical Center

    Dallas, Texas, 75390, United States

  • Virginia Commonwealth University

    Richmond, Virginia, 23298, United States

Conditions

Explore the condition pages connected to this study.