New drug shows promise for kids with duchenne muscular dystrophy

NCT ID NCT05540860

First seen Nov 12, 2025 · Last updated May 13, 2026 · Updated 26 times

Summary

This study tests a drug called sevasemten in children aged 4-9 with Duchenne muscular dystrophy. The goal is to see if it is safe and how it affects muscle health. Some children get the drug, others get a placebo, and later all can receive the drug. The study is active but not currently recruiting.

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This is a summary of the original study . Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.

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Contacts and locations

Locations

  • Arkansas Children's Hospital

    Little Rock, Arkansas, 72202, United States

  • Children's Hospital Colorado

    Aurora, Colorado, 80045, United States

  • Cincinnati Children's Hospital

    Cincinnati, Ohio, 45229, United States

  • Cook Children's Medical Center

    Fort Worth, Texas, 76104, United States

  • Kennedy Krieger Institute

    Baltimore, Maryland, 21205, United States

  • Nationwide Children's Hospital

    Columbus, Ohio, 43205, United States

  • Rare Disease Research

    Atlanta, Georgia, 30329, United States

  • UC Davis Medical Center

    Sacramento, California, 95817, United States

  • UCLA Medical Center

    Los Angeles, California, 90095, United States

  • University of Florida

    Gainesville, Florida, 32610, United States

  • University of Iowa

    Iowa City, Iowa, 52242, United States

  • University of Kansas Medical Center

    Kansas City, Kansas, 66160, United States

  • University of Massachusetts Memorial Medical Center

    Worcester, Massachusetts, 01605, United States

  • Washington University School of Medicine

    St Louis, Missouri, 63110, United States

Conditions

Explore the condition pages connected to this study.