PHENYLKETONURIA
Clinical trials for PHENYLKETONURIA explained in plain language.
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New PKU drug could loosen strict diet restrictions
Disease control OngoingThis phase 3 study tests a drug called PTC923 (sepiapterin) in 200 people with phenylketonuria (PKU), a genetic condition that requires a strict low-protein diet. The main goal is to see if the drug is safe over the long term and whether it allows people to eat more protein witho…
Matched conditions: PHENYLKETONURIA
Phase: PHASE3 • Sponsor: PTC Therapeutics • Aim: Disease control
Last updated Jun 27, 2026 14:03 UTC
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Can a One-Day allergy fix keep PKU patients on their meds?
Disease control OngoingThis study tests a rapid drug desensitization (RDD) protocol for adults with phenylketonuria (PKU) who have had allergic reactions to Palynziq. Over one day, patients receive gradually increasing doses to retrain their immune system. The goal is to see if they can safely restart …
Matched conditions: PHENYLKETONURIA
Phase: PHASE4 • Sponsor: BioMarin Pharmaceutical • Aim: Disease control
Last updated Jun 27, 2026 12:04 UTC
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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 called SAR444836 for adults with phenylketonuria (PKU), a genetic disorder that makes it hard to break down an amino acid called phenylalanine. The therapy uses a harmless virus to deliver a working copy of the missing gene. Researchers wa…
Matched conditions: PHENYLKETONURIA
Phase: PHASE1, PHASE2 • Sponsor: Sanofi • Aim: Disease control
Last updated Jun 27, 2026 07:51 UTC