New hope for duchenne: experimental drug targets muscle weakness in boys
NCT ID NCT06053814
First seen Nov 01, 2025 · Last updated May 14, 2026 · Updated 29 times
Summary
This study tests an experimental drug called NS-050/NCNP-03 in 20 boys aged 4 to 16 with Duchenne muscular dystrophy (DMD), a genetic condition that causes progressive muscle weakness. The drug aims to help muscles produce a needed protein by skipping a faulty section of the gene. The trial checks safety and how the drug works in the body over 12 weeks of increasing doses, followed by 24 weeks of open-label treatment.
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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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Alberta Children's Hospital
Calgary, Alberta, Canada
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Ankara Bilkent City Hospital
Ankara, Turkey (Türkiye)
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Ann & Robert H. Lurie Children's Hospital of Chicago
Chicago, Illinois, 60611, United States
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British Columbia Children's Hospital
Vancouver, British Columbia, Canada
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Children's Hospital Colorado
Aurora, Colorado, 80011, United States
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Children's Hospital Los Angeles
Los Angeles, California, 90027, United States
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Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, 19104, United States
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Hyogo Medical University Hospital
Nishinomiya, Hyōgo, 663-8501, Japan
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Istanbul University
Istanbul, Turkey (Türkiye)
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London Health Sciences Centre
London, Ontario, Canada
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Miyagi Children's Hospital
Sendai, Miyagi, 989-3126, Japan
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NHO Osaka Toneyama Medical Center
Toyonaka, Osaka, 560-8552, Japan
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National Center of Neurology and Psychiatry
Kodaira, Tokyo, 187-8551, Japan
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National Hospital Organization Nagara Medical Center
Nagara, Gifu, 502-8558, Japan
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Pusan National University Yangsan Hospital
Yangsan, Gyeongsangnam-do, South Korea
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Seoul National University Hospital
Seoul, South Korea
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University of Kansas Medical Center
Kansas City, Kansas, 66103, United States
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University of Pittsburgh Medical Center
Pittsburgh, Pennsylvania, 15213, United States
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Yeditepe University Kosuyolu Hospital
Istanbul, Turkey (Türkiye)
Conditions
Explore the condition pages connected to this study.