Information for Patients and families, coming soon.   Every hour of every day another baby experiences the...

Medical Professionals

Information for Medical Professionals, coming soon.   Every hour of every day another baby experiences the fate...

PKU Organizations

Information for Local PKU Organizations and/or Associations, coming soon.   Every hour of every day another baby...


Information for Policymakers, coming soon.   Every hour of every day another baby experiences the fate of...

About Us

In July 2017, a regional and several country-based PKU patient organizations from around the world made history by agreeing to form the first international association for PKU. This global entity will serve as a supportive umbrella organization to regional and national patient organizations and groups in bridging the gap for PKU worldwide.

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Why a global Organization?

The Global Association for PKU has been formed to close gaps in care for people who have PKU across the world. This means improving access to screening, treatment and seeking a cure.


PKU is a rare genetic disorder in which the body is unable to correctly metabolise an amino acid in protein, phenylalanine. Phenylalanine is a neurotoxin so left untreated people it builds up in the brain causing serious neurological symptoms including seizures and intellectual impairment.

How is PKU diagnosed?

PKU can be simply and quickly diagnosed via the “heel prick” test and if treatment begins immediately most people with this condition will live healthy lives. Currently 65% of babies born in the world miss out on this opportunity.

Recent News

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01 August 2021

Global PKU Patient Conference

Global PKU Patient Conference One World, One Vision 10th – 11th September Click here...

11 June 2018

2018 NPKUA Conference

Mark your calendars for our 5th NPKUA Conference, July 5-8, 2018 in Atlanta, GA!...

28 May 2018

Pegvaliase (PalynzigTM) has been approved

Fresh from the Press: Pegvaliase (now called PalynzigTM) has been approved by the FDA...