PHENYLKETONURIA
Clinical trials for PHENYLKETONURIA explained in plain language.
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New PKU drug enters first human safety testing
Disease control Not yet recruitingThis is an early safety study for a new oral drug called AG-181 in adults with phenylketonuria (PKU). The main goal is to see if the drug is safe and well-tolerated in 20 participants. Researchers will also measure how the drug moves through the body and its effect on blood pheny…
Matched conditions: PHENYLKETONURIA
Phase: PHASE1 • Sponsor: Agios Pharmaceuticals, Inc. • Aim: Disease control
Last updated Mar 30, 2026 14:34 UTC
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New approach aims to boost effectiveness of PKU drug
Disease control Not yet recruitingThis study is testing whether adding a medication called methotrexate to the existing PKU treatment Palynziq can help control the condition better. It aims to see if methotrexate reduces the body's immune reaction to Palynziq, potentially making the treatment more tolerable and e…
Matched conditions: PHENYLKETONURIA
Phase: PHASE4 • Sponsor: BioMarin Pharmaceutical • Aim: Disease control
Last updated Mar 30, 2026 14:29 UTC