New drug shows promise for kids with duchenne MD
NCT ID NCT05540860
First seen Nov 12, 2025 · Last updated May 22, 2026 · Updated 27 times
Summary
This study tests an experimental drug called sevasemten in 76 children aged 4-9 with Duchenne muscular dystrophy. The goal is to see if the drug is safe and can help control the disease by measuring side effects, drug levels in the blood, and muscle health markers. Participants are already on steroids and will be randomly assigned to receive the drug or a placebo, followed by an open-label phase where everyone gets the drug.
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This is a summary of
the original study
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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.
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
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Locations
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Arkansas Children's Hospital
Little Rock, Arkansas, 72202, United States
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Children's Hospital Colorado
Aurora, Colorado, 80045, United States
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Cincinnati Children's Hospital
Cincinnati, Ohio, 45229, United States
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Cook Children's Medical Center
Fort Worth, Texas, 76104, United States
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Kennedy Krieger Institute
Baltimore, Maryland, 21205, United States
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Nationwide Children's Hospital
Columbus, Ohio, 43205, United States
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Rare Disease Research
Atlanta, Georgia, 30329, United States
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UC Davis Medical Center
Sacramento, California, 95817, United States
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UCLA Medical Center
Los Angeles, California, 90095, United States
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University of Florida
Gainesville, Florida, 32610, United States
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University of Iowa
Iowa City, Iowa, 52242, United States
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University of Kansas Medical Center
Kansas City, Kansas, 66160, United States
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University of Massachusetts Memorial Medical Center
Worcester, Massachusetts, 01605, United States
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Washington University School of Medicine
St Louis, Missouri, 63110, United States
Conditions
Explore the condition pages connected to this study.