PHENYLKETONURIA (PKU)
Clinical trials for PHENYLKETONURIA (PKU) explained in plain language.
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One-Shot gene therapy tested for rare metabolic disorder
Disease control Not yet recruitingThis early-stage study is testing a single injection of an experimental gene therapy called GS1168 in adults with a genetic condition called PKU. The goal is to see if the treatment is safe and if it can help the body better process protein, potentially reducing the need for a st…
Matched conditions: PHENYLKETONURIA (PKU)
Phase: EARLY_PHASE1 • Sponsor: Gritgen Therapeutics Co., Ltd. • Aim: Disease control
Last updated Mar 27, 2026 12:38 UTC
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Scientists probe the hidden mental toll of PKU in adults
Knowledge-focused Not yet recruitingThis study aims to understand how different levels of phenylalanine in the blood affect the thinking, emotions, and daily life of adults with PKU. Researchers will observe 30 adults with PKU and a close relative to learn more about their personal experiences. The goal is to gathe…
Matched conditions: PHENYLKETONURIA (PKU)
Sponsor: Central Hospital, Nancy, France • Aim: Knowledge-focused
Last updated Mar 27, 2026 12:40 UTC
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Scientists hunt genetic clues to explain why PKU affects adults differently
Knowledge-focused Not yet recruitingThis study aims to understand why adults with PKU, a genetic metabolic disorder, experience the disease so differently from one another. Researchers will analyze DNA from saliva and blood samples from about 150 adults with PKU to look for links between their genes, their metaboli…
Matched conditions: PHENYLKETONURIA (PKU)
Sponsor: University Hospital, Tours • Aim: Knowledge-focused
Last updated Mar 23, 2026 15:18 UTC