The overall aim of this study is to evaluate LNAA treatment as a potential alternative to conventional dietary treatment for PKU. This study investigates the effects of LNAA treatment compared to the classic dietary treatment on cerebral dopamine synthesis in patients with classic PKU. We will assess LNAAs effectiveness on neurotransmitter synthesis, cognitive function, mental health, and safety, compared to the standard diet.
Treatment initiation within the first month of life
Intelligence quotient over 84, based upon the baseline neuropsychological evaluation
Conventional dietary treatment up to minimum 15 years of age
Signed informed consent
Unable or unwilling to adhere to the requirements of the study
A female who is pregnant or breastfeeding or planning to get pregnant during the study period
Concomitant medication that may interfere with the PET analysis, as judged by the investigator
A serious neuropsychiatric disease that could interfere with the subject's ability to participate in the study at the discretion of the investigator
Rigshospitalet, Denmark
Lead sponsor
Prekulab Ltd ApS
Collaborator