Researchers track rare disorder in kids to unlock genetic clues

NCT ID NCT06723925

Summary

This study aims to better understand biotinidase deficiency, a rare inherited disorder, in children identified through newborn screening in Italy. Researchers will follow about 180 children for at least three years to see how their specific genetic changes relate to their symptoms and response to treatment. The goal is to gather knowledge to improve future care, not to test a new therapy.

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Contacts and locations

Study contacts

  • Contact

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

Locations

  • IRCCS Azienda Ospedaliero-Universitaria di Bologna

    RECRUITING

    Bologna, Bologna, 40138, Italy

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

Conditions

Explore the condition pages connected to this study.