Mr Edwards, from Llanegryn in Gwynedd, was diagnosed with PKU at birth after it showed up in the standard blood tests given to babies - the condition affects one in 10,000 people in the UK.

People affected by phenylketonuria (PKU) unite to describe challenges of managing PKU and aspirations for the future of PKU treatment WASHINGTON, Dec. 5, 2024 /PRNewswire/ -- The National PKU ...

Patients in the Phearless study of BMN 307 – an adeno-associated virus (AAV) gene therapy designed to replace the phenylalanine hydroxylase (PAH) deficient in people with PKU – are getting a ...