PHENYLKETONURIA (PKU)
Clinical trials for PHENYLKETONURIA (PKU) explained in plain language.
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New shot could free teens with PKU from strict diet
Disease control OngoingThis study tests an injectable drug called pegvaliase in 55 teenagers (ages 12-17) with phenylketonuria (PKU) who have high blood Phe levels despite diet management. Half get the drug, half stick to diet only. The goal is to see if pegvaliase safely lowers Phe levels and reduces …
Matched conditions: PHENYLKETONURIA (PKU)
Phase: PHASE3 • Sponsor: BioMarin Pharmaceutical • Aim: Disease control
Last updated Jun 26, 2026 16:51 UTC
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48-Hour test may predict who benefits from PKU drug
Knowledge-focused OngoingThis study tests whether a 48-hour BH4 loading test can predict which people with phenylketonuria (PKU) will respond to treatment. Twenty participants receive BH4 and have their blood phenylalanine levels measured over two days. The goal is to link test results with each person's…
Matched conditions: PHENYLKETONURIA (PKU)
Phase: PHASE1 • Sponsor: Sohag University • Aim: Knowledge-focused
Last updated Jun 27, 2026 08:00 UTC