PHENYLKETONURIAS
Clinical trials for PHENYLKETONURIAS explained in plain language.
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One-Shot gene therapy aims to fix rare genetic disorder
⭐️ CURE ⭐️ Recruiting nowThis early-stage study is testing a new one-time gene therapy called NGGT002 for adults with classic phenylketonuria (PKU). The goal is to see if delivering a working copy of the faulty gene is safe and can lower harmful blood levels of phenylalanine, potentially reducing the nee…
Matched conditions: PHENYLKETONURIAS
Phase: PHASE1, PHASE2 • Sponsor: NGGT INC. • Aim: ⭐️ CURE ⭐️
Last updated Mar 13, 2026 15:05 UTC
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New PKU drug study aims to unlock a more normal diet and life
Disease control Recruiting nowThis study is observing adults with PKU who are starting a new, FDA-approved drug called Palynziq. Researchers want to see how this treatment affects what people can eat, their brain function, nutritional status, and overall quality of life over three to five years. The goal is t…
Matched conditions: PHENYLKETONURIAS
Sponsor: Emory University • Aim: Disease control
Last updated Mar 27, 2026 12:40 UTC
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One-Shot gene therapy trial offers hope for lifelong PKU management
Disease control Recruiting nowThis early-stage study is testing a single intravenous infusion of an experimental gene therapy called NGGT002 in adults with phenylketonuria (PKU). The main goals are to see if the treatment is safe and if it can lower the high levels of phenylalanine (Phe) in the blood, which i…
Matched conditions: PHENYLKETONURIAS
Phase: EARLY_PHASE1 • Sponsor: The First Affiliated Hospital of Bengbu Medical University • Aim: Disease control
Last updated Mar 27, 2026 12:37 UTC
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New video therapy aims to ease mental health struggles for teens with rare disorder
Symptom relief Recruiting nowThis study is testing whether a 10-week video therapy program can help teenagers with PKU manage anxiety and depression. Researchers will enroll 30 adolescents aged 10-17 who have PKU and experience these symptoms. Half will start therapy immediately, while the other half will wa…
Matched conditions: PHENYLKETONURIAS
Phase: NA • Sponsor: University of Missouri-Columbia • Aim: Symptom relief
Last updated Mar 12, 2026 13:50 UTC