PHENYLKETONURIA (PKU)
Clinical trials for PHENYLKETONURIA (PKU) explained in plain language.
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One-Shot gene therapy could ease PKU diet burden
Disease control Not yet recruitingThis early-stage study tests a single injection of a gene therapy (GS1168) in 9 adults with classical PKU whose condition is not well controlled by diet or other treatments. The goal is to see if the therapy is safe and can lower harmful phenylalanine levels in the blood, potenti…
Matched conditions: PHENYLKETONURIA (PKU)
Phase: EARLY_PHASE1 • Sponsor: Gritgen Therapeutics Co., Ltd. • Aim: Disease control
Last updated May 12, 2026 13:39 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 affect thinking, emotions, and daily functioning in 30 adults with PKU. Researchers want to understand why some patients with high Phe levels have no major problems, while others do. The goal is to move toward personalized …
Matched conditions: PHENYLKETONURIA (PKU)
Sponsor: Central Hospital, Nancy, France • Aim: Knowledge-focused
Last updated May 15, 2026 11:56 UTC