PHENYLKETONURIA (PKU)
Clinical trials for PHENYLKETONURIA (PKU) explained in plain language.
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One-Shot gene therapy aims to ease PKU diet burden
Disease control Not yet recruitingThis early study tests a single injection of a gene therapy (GS1168) in 9 adults with classic PKU that is not well controlled by diet or other treatments. The goal is to see if it is safe and can lower harmful phenylalanine levels in the blood, possibly allowing a more normal die…
Matched conditions: PHENYLKETONURIA (PKU)
Phase: EARLY_PHASE1 • Sponsor: Gritgen Therapeutics Co., Ltd. • Aim: Disease control
Last updated May 26, 2026 10:10 UTC
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PKU mystery: why do some patients thrive despite high phe levels?
Knowledge-focused Not yet recruitingThis study looks at how blood phenylalanine (Phe) levels impact the thinking, emotions, and daily functioning of 30 adults with PKU. Researchers want to understand why some patients with high Phe levels have no major brain problems. No treatment is given—only questionnaires and o…
Matched conditions: PHENYLKETONURIA (PKU)
Sponsor: Central Hospital, Nancy, France • Aim: Knowledge-focused
Last updated May 26, 2026 10:13 UTC