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PKU brain study reveals how new treatment may boost thinking skills

NCT ID NCT05356377

First seen Jan 06, 2026 · Last updated Jun 12, 2026 · Updated 29 times

Summary

This study looked at how the drug Palynziq, which lowers phenylalanine levels in the blood, affects the brain and thinking abilities in adults with phenylketonuria (PKU). Six participants who were already taking Palynziq underwent brain scans and cognitive tests to measure changes in brain structure and function. The goal was to understand the neurological improvements linked to long-term reduction of phenylalanine levels.

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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

  • Boston Children's Hospital

    Boston, Massachusetts, 02115, United States

  • University of Missouri

    Columbia, Missouri, 65211, United States

Conditions

The condition(s) this trial relates to.

phenylketonuria

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.