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Decade-Long safety watch for groundbreaking duchenne gene therapy

NCT ID NCT05689164

Summary

This study follows boys with Duchenne muscular dystrophy for 10 years after they received an experimental gene therapy in previous Pfizer trials. The goal is to monitor long-term safety and see how well the treatment continues to work over time. Participants will have annual check-ins to track their health, mobility, and heart function.

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

  • Duke Children's Health Center

    Durham, North Carolina, 27710, United States

  • Duke Lenox Baker Children's

    Durham, North Carolina, 27705, United States

  • Primary Children's Hospital

    Salt Lake City, Utah, 84113, United States

  • Reed Neurological Research Center

    Los Angeles, California, 90095, United States

  • UCLA Children's Heart Center

    Los Angeles, California, 90095, United States

  • UCLA Clinical Lab Services

    Los Angeles, California, 90095, United States

  • UCLA Kameron Gait and Motion Analysis Laboratory (Westwood Rehabilitation Center)

    Los Angeles, California, 90095, United States

  • UCLA Medical Center

    Los Angeles, California, 90095, United States

  • University of Utah Clinical Neurosciences Center

    Salt Lake City, Utah, 84132, United States

  • University of Utah Craig H. Neilsen Rehabilitation Hospital

    Salt Lake City, Utah, 84132, United States

  • University of Utah Hospital

    Salt Lake City, Utah, 84112, United States

  • University of Utah Imaging and Neurosciences Center

    Salt Lake City, Utah, 84108, United States

Conditions

Explore the condition pages connected to this study.