Landmark study seeks to unlock mysteries of rare metabolic disease

NCT ID NCT00078078

Summary

This study aims to better understand methylmalonic acidemia (MMA), a rare and serious inherited metabolic disorder. Researchers will observe over 2,000 patients to learn how the disease progresses, identify complications, and find biological markers. The goal is to gather essential information to help design future treatment trials, not to test a new therapy directly.

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: •••••@•••••

  • Contact

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

Locations

  • Children's National Medical Center

    COMPLETED

    Washington D.C., District of Columbia, 20010, United States

  • National Institutes of Health Clinical Center

    RECRUITING

    Bethesda, Maryland, 20892, United States

  • UPMC Children's Hospital of Pittsburgh

    RECRUITING

    Pittsburgh, Pennsylvania, 15224, United States

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

Conditions

Explore the condition pages connected to this study.