PHENYLKETONURIA (PKU)
Clinical trials for PHENYLKETONURIA (PKU) explained in plain language.
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New injection offers hope for teens with rare genetic disorder
Disease control OngoingThis study is testing whether daily injections of a medication called pegvaliase are safe and effective for teenagers with PKU, a genetic disorder that makes it hard to process protein. About 55 teens will be randomly assigned to receive either the injections or continue managing…
Matched conditions: PHENYLKETONURIA (PKU)
Phase: PHASE3 • Sponsor: BioMarin Pharmaceutical • Aim: Disease control
Last updated Mar 18, 2026 14:41 UTC
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Scientists search for clues in genes to personalize PKU treatment
Knowledge-focused OngoingThis study aims to understand how patients with Phenylketonuria (PKU) respond to a supplement called tetrahydrobiopterin (BH4). Researchers will give BH4 to 20 patients and measure changes in a key blood chemical. They will also check if a patient's genetic makeup can predict how…
Matched conditions: PHENYLKETONURIA (PKU)
Phase: PHASE1 • Sponsor: Sohag University • Aim: Knowledge-focused
Last updated Apr 01, 2026 14:43 UTC