PHENYLKETONURIAS
Clinical trials for PHENYLKETONURIAS explained in plain language.
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One-Time gene infusion aims to tame PKU for years
Disease control Recruiting nowThis early-phase trial tests a single intravenous dose of NGGT002, a gene therapy for adults with phenylketonuria (PKU). PKU is a genetic condition that causes harmful buildup of phenylalanine (Phe) in the blood, requiring a strict low-protein diet. The study will enroll 15 parti…
Matched conditions: PHENYLKETONURIAS
Phase: EARLY_PHASE1 • Sponsor: The First Affiliated Hospital of Bengbu Medical University • Aim: Disease control
Last updated May 17, 2026 11:57 UTC
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New drug lets PKU patients eat more protein – but lifelong treatment still needed
Disease control Recruiting nowThis study looks at how Palynziq, a medication for phenylketonuria (PKU), improves diet, brain function, and nutrition. PKU is a genetic disorder that makes it hard to break down an amino acid called phenylalanine, which can build up and cause brain damage. The study will follow …
Matched conditions: PHENYLKETONURIAS
Sponsor: Emory University • Aim: Disease control
Last updated May 15, 2026 11:57 UTC
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New gene therapy hopes to ease PKU's strict diet for life
Disease control Recruiting nowThis early-stage trial tests a one-time gene therapy called NGGT002 in 12 adults with classic PKU, a genetic disorder that makes it hard to break down an amino acid called phenylalanine. The therapy delivers a working copy of the PAH gene to help the body process phenylalanine no…
Matched conditions: PHENYLKETONURIAS
Phase: PHASE1, PHASE2 • Sponsor: NGGT INC. • Aim: Disease control
Last updated May 01, 2026 15:57 UTC
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New therapy aims to ease anxiety in teens with rare metabolic disorder
Symptom relief Recruiting nowThis study tests a short, skills-based therapy program (Show Me FIRST) to help reduce anxiety and depression in 30 adolescents (ages 10-17) with PKU, a rare genetic condition. Participants are randomly assigned to start therapy right away or after a waiting period. The program fo…
Matched conditions: PHENYLKETONURIAS
Phase: NA • Sponsor: University of Missouri-Columbia • Aim: Symptom relief
Last updated May 15, 2026 11:54 UTC