New hope for duchenne: experimental drug BMN 351 enters human testing

NCT ID NCT06280209

First seen Feb 03, 2026 · Last updated May 10, 2026 · Updated 9 times

Summary

This study tests a new medicine, BMN 351, in 18 boys aged 4 to 10 with Duchenne muscular dystrophy who have a specific genetic change. The main goal is to check if the drug is safe and how the body handles it. Participants receive multiple doses through an IV, and researchers will monitor side effects and drug levels over about 97 weeks.

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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

  • 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

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