Can a new drug protect the developing brain in kids with PKU?
NCT ID NCT06302348
Summary
This study aims to see if long-term treatment with a drug called sepiapterin can help preserve normal brain development and thinking skills in children with phenylketonuria (PKU). It will enroll 56 children, from toddlers up to age 16, who have PKU. Researchers will track changes in intelligence scores and quality of life over time to see if the treatment helps control the disease and protect cognitive function.
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Study contacts
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Contact
Phone: •••-•••-•••• Email: •••••@•••••
Locations
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Indiana University
RECRUITINGIndianapolis, Indiana, 46202, United States
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