PHENYLKETONURIA
Clinical trials for PHENYLKETONURIA explained in plain language.
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One-Time gene therapy could free PKU patients from strict diet
Disease control OngoingThis study tests a one-time gene therapy (SAR444836) for adults with phenylketonuria (PKU), a genetic disorder that makes it hard to break down an amino acid called phenylalanine (Phe). The goal is to lower Phe levels in the blood and allow participants to eat a normal diet witho…
Matched conditions: PHENYLKETONURIA
Phase: PHASE1, PHASE2 • Sponsor: Sanofi • Aim: Disease control
Last updated May 17, 2026 10:08 UTC
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New pill for PKU shows promise in Long-Term safety trial
Disease control OngoingThis study tests the long-term safety of an experimental drug called PTC923 (sepiapterin) in 200 people with phenylketonuria (PKU), a genetic condition that makes it hard to process the amino acid phenylalanine. Participants take the drug and researchers track side effects and ch…
Matched conditions: PHENYLKETONURIA
Phase: PHASE3 • Sponsor: PTC Therapeutics • Aim: Disease control
Last updated May 17, 2026 10:04 UTC
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New protocol aims to stop allergic reactions to PKU drug
Symptom relief OngoingThis study tests a one-day rapid drug desensitization (RDD) procedure in 9 adults with phenylketonuria (PKU) who have had allergic reactions to their medication Palynziq. The goal is to see if RDD allows them to restart and continue their regular dose without further severe react…
Matched conditions: PHENYLKETONURIA
Phase: PHASE4 • Sponsor: BioMarin Pharmaceutical • Aim: Symptom relief
Last updated May 11, 2026 20:46 UTC