Gene therapy injection aims to restore swallowing in rare muscle disorder

NCT ID NCT06185673

First seen Jan 05, 2026 · Last updated Apr 29, 2026 · Updated 14 times

Summary

This study tests a single injection of BB-301 into throat muscles to improve swallowing in people with oculopharyngeal muscular dystrophy (OPMD). About 30 adults aged 50-65 who have already been in a natural history study will receive the treatment during a short surgery. The main goals are to check safety and find the best dose, while also measuring how well it reduces food sticking in the throat.

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Contacts and locations

Study contacts

  • Contact

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

  • Contact

    Email: •••••@•••••

Locations

  • NYU Langone Health

    RECRUITING

    New York, New York, 10016, United States

    Contact

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

Conditions

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