With their benchmark report in 2012, the European patient organization E.S.PKU has identified significant inconsistencies in PKU treatment and access to care in Europe, and called for uniform guidelines. Coordinated by the Chairman of the Scientific Advisory Committee of the E.S.PKU, since then a group of renowned metabolic experts independently developed 70 statements based on available evidence and taking into account nearly 500 scientific publications. Prof. van Spronsen stated: “Remaining within national frameworks is now outdated practice.”
At the beginning of 2017 the ten key statements of the European guidelines were already published in Lancet Diabetes, Endocrinology and Metabolism. With the present publication in the OJRD, the entire treatment spectrum of phenylketonuria is considered. Eric Lange, President of ESPKU says: “PKU knows no borders. These comprehensive guidelines are fundamental for a fairer and more consistent access to good treatment quality for all patients, despite their origin.”
However, the mission is not accomplished: “We invite all healthcare providers to immediately implement into daily practice and to give patients access to a state of the art treatment”, added van Spronsen. A first update of the guidelines is already being prepared to include latest scientific findings and innovative treatments.
Start reading right now, click the image to get to the Orphanet Journal of Rare Diseases! If you want to learn more about the guidelines you can visit our dedicated page.
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