PHENYLKETONURIA
Clinical trials for PHENYLKETONURIA explained in plain language.
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One-Shot gene therapy aims to free PKU patients from lifelong diet
⭐️ CURE ⭐️ OngoingThis study is testing a new, one-time gene therapy injection for adults with phenylketonuria (PKU). The goal is to see if the treatment is safe and if it can lower harmful blood Phe levels enough to allow participants to stop their highly restrictive, lifelong low-protein diet. R…
Matched conditions: PHENYLKETONURIA
Phase: PHASE1, PHASE2 • Sponsor: Sanofi • Aim: ⭐️ CURE ⭐️
Last updated Mar 30, 2026 14:29 UTC
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New PKU treatment could ease strict diet rules
Disease control OngoingThis study is testing the long-term safety of an investigational medication called PTC923 for people with phenylketonuria (PKU). It involves 200 participants and aims to see if the treatment allows people to safely eat more protein while keeping their blood phenylalanine levels i…
Matched conditions: PHENYLKETONURIA
Phase: PHASE3 • Sponsor: PTC Therapeutics • Aim: Disease control
Last updated Mar 23, 2026 15:16 UTC