Real-World data sought on DMD Exon-Skipping drugs

NCT ID NCT06606340

ENROLLING_BY_INVITATION Knowledge-focused Sponsor: Sarepta Therapeutics, Inc. Source: ClinicalTrials.gov ↗

First seen Jun 24, 2026 · Last updated Jun 26, 2026 · Updated 1 time

Summary

This observational study will follow 300 people with Duchenne muscular dystrophy who are already taking exon-skipping therapies (eteplirsen, golodirsen, or casimersen) as part of their routine care. Researchers will collect data on movement, lung function, and heart function over time to see how these treatments work in everyday practice. No new drugs are being tested; the goal is to gather long-term safety and effectiveness information.

What this could mean

Our plain-language read of the trial. This is informational only — not medical advice or a prediction.

Active substance: eteplirsen, golodirsen, casimersen
What this could lead to: If successful, this study could provide real-world evidence on how well these exon-skipping therapies work over the long term for Duchenne muscular dystrophy.
What could go wrong: This is an observational study, not a controlled trial, so results may be influenced by factors like differences in patient care. It does not test new treatments, only monitors existing ones.

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Conditions

The condition(s) this trial relates to.

Duchenne muscular dystrophy

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

DUCHENNE MUSCULAR DYSTROPHY

Contacts and locations

Locations

Arkansas Children's Hospital Research Institute

Little Rock, Arkansas, 72202, United States
Atrium Health Wake Forest University Health Sciences

Winston-Salem, North Carolina, 27157, United States
Children's Hospital Los Angeles

Los Angeles, California, 90027, United States
Children's Hospital Medical Center

Cincinnati, Ohio, 45229, United States
Children's Hospital of Philadelphia

Philadelphia, Pennsylvania, 19104, United States
Children's National Hospital

Washington D.C., District of Columbia, 20010, United States
Columbia University Medical Center - PIN

New York, New York, 10032, United States
Cook Children's Hospital

Fort Worth, Texas, 76104, United States
Duke Lenox Baker Children's Hospital

Durham, North Carolina, 27705, United States
Kennedy Krieger Institute

Baltimore, Maryland, 21287, United States
Nationwide Children's Hospital

Columbus, Ohio, 43205, United States
Penn State Health Milton S. Hershey Medical Center

Hershey, Pennsylvania, 17033-0850, United States
Seattle Children's Hospital

Seattle, Washington, 98105, United States
Texas Children's Hospital

Houston, Texas, 77030, United States
UPMC Children's Hospital of Pittsburgh

Pittsburgh, Pennsylvania, 15213, United States
UVA Children's Hospital

Charlottesville, Virginia, 22908, United States
University Of Iowa Hospitals And Clinics

Iowa City, Iowa, 52242, United States
University of California Davis Medical Center

Sacramento, California, 95817, United States
University of Florida

Gainesville, Florida, 32610, United States
Washington University

St Louis, Missouri, 63110, United States

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