First human test: can mRNA fix broken muscles in DMD kids?

NCT ID NCT07188012

Summary

This early study is testing whether a new treatment called SPOT-mRNA03 is safe for boys with Duchenne muscular dystrophy (DMD). The treatment delivers genetic instructions to help muscles produce a missing protein called dystrophin. Six boys aged 2-6 will receive the treatment through IV infusions while researchers monitor for side effects and check if their muscles start making the needed protein.

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Contacts and locations

Study contacts

  • Contact

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

Locations

  • Shanghai Children's Medical Center

    RECRUITING

    Shanghai, Shanghai Municipality, China

    Contact

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

Conditions

Explore the condition pages connected to this study.