PHENYLKETONURIA (PKU)
Clinical trials for PHENYLKETONURIA (PKU) explained in plain language.
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Shot of hope for teens with PKU: new drug may ease strict diet burden
Disease control OngoingThis study tests a medicine called pegvaliase, given as a shot under the skin, in teenagers aged 12-17 with phenylketonuria (PKU). The goal is to see if it safely lowers high blood phenylalanine levels better than a strict diet alone. About 55 teens will take part, with half gett…
Matched conditions: PHENYLKETONURIA (PKU)
Phase: PHASE3 • Sponsor: BioMarin Pharmaceutical • Aim: Disease control
Last updated May 18, 2026 12:02 UTC
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Quick test may predict PKU drug success
Knowledge-focused OngoingThis study looked at 20 people with phenylketonuria (PKU) to see if a 48-hour test with BH4 (a supplement) could predict who would benefit from long-term treatment. Participants took BH4 and had blood samples taken over two days. The goal was to see if a drop in phenylalanine lev…
Matched conditions: PHENYLKETONURIA (PKU)
Phase: PHASE1 • Sponsor: Sohag University • Aim: Knowledge-focused
Last updated May 22, 2026 13:51 UTC