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New drug tested for boys with Muscle-Wasting disease

NCT ID NCT06280209

Summary

This early-stage study is testing a new drug called BMN 351 in boys with Duchenne muscular dystrophy who have a specific genetic mutation. The main goal is to check if the drug is safe and to find the right dose when given through an IV. About 18 boys aged 4-10 who can walk without assistance will receive different doses over time while researchers monitor their health.

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

  • Children's Hospital LHSC

    RECRUITING

    London, Ontario, N6A 5W9, Canada

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

    Contact

  • Fondazione Serena ETS - Centro Clinico NeMO Milano

    RECRUITING

    Milan, Italy

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

    Contact

  • Great Ormond Street Hospital NHS Foundation Trust

    RECRUITING

    London, WC1N 3JH, United Kingdom

    Contact

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

  • Hospital Sant Joan de Deu

    RECRUITING

    Barcelona, 08950, Spain

    Contact

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

  • Hospital Viamed Santa Angela De la Cruz

    RECRUITING

    Seville, 41013, Spain

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

    Contact

  • Leids Universitair Medisch Centrum

    RECRUITING

    Leiden, 2333 ZA, Netherlands

    Contact

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

  • UOC Fase I - Fondazione Policlinico Universitario A. Gemelli IRCCS - Universita Cattolica del Sacro Cuore

    RECRUITING

    Rome, Italy

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

    Contact

  • Yeditepe University Kosuyolu Hospital

    RECRUITING

    Istanbul, Turkey (Türkiye)

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

    Contact

Conditions

Explore the condition pages connected to this study.