Hope for rare disease patients: long-term treatment study continues

NCT ID NCT05295433

Summary

This study continues testing mRNA-3705 in people with methylmalonic acidemia (MMA), a rare genetic disorder. It follows patients who previously received this experimental treatment in earlier studies to check long-term safety and effectiveness. The goal is to see if continued treatment helps control the disease over time.

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

Study contacts

  • Contact

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

Locations

  • Altman Clinical and Translational Research Institution

    RECRUITING

    San Diego, California, 92037, United States

    Contact Email: •••••@•••••

  • Birmingham Children's Hospital NHS Foundation Trust

    TERMINATED

    Birmingham, B4 6NH, United Kingdom

  • Erasmus MC

    RECRUITING

    Rotterdam, 3015 AA, Netherlands

    Contact Email: •••••@•••••

    Contact

  • Hospital For Sick Children

    RECRUITING

    Toronto, Ontario, M5G 1X8, Canada

    Contact Email: •••••@•••••

  • Hospital Universitario 12 de Octubre

    RECRUITING

    Madrid, 28041, Spain

    Contact Email: •••••@•••••

  • Hospital Universitario Cruces

    RECRUITING

    Barakaldo, Vizcaya, 48903, Spain

    Contact Email: •••••@•••••

  • Hôpital Necker - Enfants Malades APHP

    RECRUITING

    Paris, 75015, France

    Contact Email: •••••@•••••

  • Lucile Packard Children's Hospital at Stanford

    RECRUITING

    Palo Alto, California, 94304, United States

    Contact Email: •••••@•••••

  • Royal Manchester Childrens Hospital

    RECRUITING

    Manchester, M13 9WL, United Kingdom

    Contact Email: •••••@•••••

  • Stollery Children's Hospital University of Alberta

    RECRUITING

    Edmonton, Alberta, T6G 2R7, Canada

    Contact Email: •••••@•••••

  • UCLA Medical Center

    RECRUITING

    Los Angeles, California, 90095, United States

    Contact Email: •••••@•••••

  • Universitair Medisch Centrum Utrecht

    RECRUITING

    Utrecht, 3584 CX, Netherlands

    Contact Email: •••••@•••••

    Contact

Conditions

Explore the condition pages connected to this study.