New hope for boys with DMD: weekly infusion aims to restore muscle protein

NCT ID NCT05996003

Summary

This study is testing a new drug called NS-089/NCNP-02 for boys with Duchenne muscular dystrophy (DMD) caused by a specific genetic error (exon 44). The drug is given as a weekly IV infusion and aims to help muscles produce more of a crucial protein called dystrophin. Researchers will measure if the drug is safe, how the body processes it, and if it improves muscle function and strength in 20 boys aged 4 to 15 who can still walk.

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

Study contacts

  • Contact

    Phone: •••-•••-•••• Email: •••••@•••••

Locations

  • Alberta Children's Hospital

    NOT_YET_RECRUITING

    Calgary, Alberta, Canada

    Contact Email: •••••@•••••

  • Ankara Bilkent City Hospital

    NOT_YET_RECRUITING

    Ankara, 06800, Turkey (Türkiye)

    Contact Email: •••••@•••••

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

    RECRUITING

    Chicago, Illinois, 60611, United States

    Contact Email: •••••@•••••

    Contact Email: •••••@•••••

  • British Columbia Children's Hospital

    NOT_YET_RECRUITING

    Vancouver, British Columbia, Canada

    Contact Email: •••••@•••••

  • Children's Hospital Colorado

    RECRUITING

    Aurora, Colorado, 80045, United States

    Contact Email: •••••@•••••

  • Cincinnati Children's Hospital Medical Center

    RECRUITING

    Cincinnati, Ohio, 45229, United States

    Contact Email: •••••@•••••

  • Fukui Prefectural Hospital

    NOT_YET_RECRUITING

    Fukui-shi, Fukui, 910-8526, Japan

  • London Health Sciences Centre

    RECRUITING

    London, Ontario, Canada

    Contact Email: •••••@•••••

  • NHO Osaka Toneyama Medical Center

    NOT_YET_RECRUITING

    Toyonaka, Osaka, 560-8552, Japan

    Contact Email: •••••@•••••

  • National Center of Neurology and Psychiatry

    NOT_YET_RECRUITING

    Kodaira, Tokyo, 187-8551, Japan

  • National Hospital Organization Nagara Medical Center

    RECRUITING

    Nagara, Gifu-shi, Gifu, 502-8558, Japan

    Contact Email: •••••@•••••

  • Perth Children's Hospital

    NOT_YET_RECRUITING

    Nedlands, Western Australia, 6009, Australia

    Contact Email: •••••@•••••

  • Pusan National University Yangsan Hospital

    NOT_YET_RECRUITING

    Yangsan, Gyeongsangnam, South Korea

    Contact Email: •••••@•••••

  • Rare Disease Research

    RECRUITING

    Atlanta, Georgia, 30329, United States

    Contact Email: •••••@•••••

  • Rare Disease Research, LLC - FL

    RECRUITING

    Kissimmee, Florida, 34746, United States

    Contact Email: •••••@•••••

  • S.B.U. Dr. Behcet uz Pediatric Diseases and Surgery Training and Research Hospital

    NOT_YET_RECRUITING

    Izmir, 11794, Turkey (Türkiye)

    Contact Email: •••••@•••••

  • Seoul National University Hospital

    NOT_YET_RECRUITING

    Seoul, South Korea

    Contact Email: •••••@•••••

  • Shiga General Hospital

    NOT_YET_RECRUITING

    Moriyama-shi, Shiga, 524-8524, Japan

    Contact Email: •••••@•••••

  • Starship Children's Hospital

    NOT_YET_RECRUITING

    Auckland, 1023, New Zealand

    Contact Email: •••••@•••••

  • The Children's Hospital of Philadelphia (CHOP)

    RECRUITING

    Philadelphia, Pennsylvania, 19104, United States

    Contact Email: •••••@•••••

  • UT Southwestern/Children's Health

    RECRUITING

    Dallas, Texas, 75207, United States

    Contact Email: •••••@•••••

  • University of Kansas Medical Center (KUMC)

    RECRUITING

    Kansas City, Kansas, 66160, United States

    Contact Email: •••••@•••••

  • University of Pittsburgh School of Medicine

    RECRUITING

    Pittsburgh, Pennsylvania, 15224, United States

    Contact Email: •••••@•••••

  • Virginia Commonwealth University Health System

    RECRUITING

    Richmond, Virginia, 23298, United States

    Contact Email: •••••@•••••

  • Yeditepe University Kosuyolu Hospital

    NOT_YET_RECRUITING

    Istanbul, 34718, Turkey (Türkiye)

    Contact Email: •••••@•••••

Conditions

Explore the condition pages connected to this study.