Phenylketonuria Round Table at the EU Parliament

Prof Maurizio Scarpa (MetabERN Coordinator), Francjan van Spronsen (MetabERN Representative from Rijksuniversiteit Groningen (NL)), and Lut de Baere (responsible for the MetabERN Patients organizations management), have participated in the policy Round Table on Phenylketonuria (PKU) “‘Addressing the high burden and significant unmet needs in the management of PKU’” at the European Parliament on 11th July 2018 (see attached agenda).

The event, co-hosted by MEPs Nessa Childers and Deirdre Clune and organized in collaboration with ESPKU, was aimed to raise awareness of PKU, address ways to better manage the condition and improve patient outcomes.

In his talk on “The journey towards improving patient outcomes: What has been achieved?” Francjan highlighted the important role of MetabERN in providing a cross-sectorial environment in which inter-professional collaborative teams composed by health-care professionals and patient organizations cooperatively working together in a common collaborative effort aimed to address patient’s needs.

MetabERN facilitates cross border collaboration helping patients, families and carers find common fair, equitable and equal solutions across EU and reminding them they are not alone.

The Roundtable brought together different stakeholders with common interest in addressing unmet needs in the field of PKU, i.e. patients’ and health professionals’ organisations (psychologists, GP’s, nurses, neurologists, paediatricians, dieticians….), carers, social workers, payers and policy makers.

Phenylketonuria (PKU) is an inherited metabolic disorder that affects about one in every 10,000 children born in Europe. If left untreated, PKU results in increased phenylalanine concentrations in blood and brain. This can cause severe intellectual disability, epilepsy and behavioural problems. In Europe, the mean prevalence of the condition is approximately 1: 10,000 newborns.

PKU is increasingly recognised by policymakers as a condition which needs attention. However, continued efforts are required to keep the topic on the EU and national policy agendas, and this is what the Roundtable aims to achieve.