PHENYLKETONURIA (PKU)
Clinical trials for PHENYLKETONURIA (PKU) explained in plain language.
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Shot of hope: new drug may free teens with PKU from strict diet
Disease control OngoingThis study tests a drug called pegvaliase, given as a shot under the skin, in teenagers aged 12-17 with phenylketonuria (PKU) who have trouble keeping their blood Phe levels under control with diet alone. About 55 teens will be randomly assigned to either get the drug or stick wi…
Matched conditions: PHENYLKETONURIA (PKU)
Phase: PHASE3 • Sponsor: BioMarin Pharmaceutical • Aim: Disease control
Last updated May 11, 2026 20:46 UTC
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Quick test may unlock personalized PKU care
Knowledge-focused OngoingThis study looks at whether a 48-hour test with a substance called BH4 can predict how well people with phenylketonuria (PKU) will respond to treatment. Researchers gave 20 participants BH4 and measured their blood phenylalanine levels over two days. They also checked if the resp…
Matched conditions: PHENYLKETONURIA (PKU)
Phase: PHASE1 • Sponsor: Sohag University • Aim: Knowledge-focused
Last updated May 14, 2026 12:06 UTC