Den här översättningen är inte klar ännu. Den här sidan är just nu på engelska.

Gå till den engelska sidan

One-shot gene therapy aims to give duchenne boys stronger muscles

NCT ID NCT07160634

First seen Oct 31, 2025 · Last updated May 20, 2026 · Updated 33 times

Summary

This study tests a single intravenous dose of SGT-003, a gene therapy, in 80 boys with Duchenne muscular dystrophy who can still walk. The goal is to see if it improves muscle function, like how fast they can stand up or climb stairs. Participants will receive either the therapy or a placebo first, then switch, and be followed for at least 5 years.

Disclaimer Read more

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.

Get updates

Get notified about this study

Sign up to get updates when this study changes or when new studies for DUCHENNE MUSCULAR DYSTROPHY are added.

Vår säkerhetsrekommendation!

Genom att skicka in godkänner du våra Användarvillkor

Contacts and locations

Study contacts

  • Contact

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

Locations

  • BC Children's Hospital

    RECRUITING

    Vancouver, British Columbia, Canada

    Contact

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

  • The Children's Hospital of Westmead

    RECRUITING

    Sydney, New South Wales, Australia

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

    Contact

Conditions

Explore the condition pages connected to this study.