Pegvaliase (PalynzigTM) has been approved

Fresh from the Press: Pegvaliase (now called PalynzigTM) has been approved by the FDA for use in adult PKU patients in the USA with phenylalanine > 600 micromol/l. Francjan J van Spronsen, Maria Gizewska, Anita MacDonald, Kirsten Ahring   Why is a different treatment needed? ‘You are not lucky when you have PKU’ I explained to a colleague. He replied “Why? What you’ve always told me is that when left untreated, patients with PKU have severe developmental disability, epilepsy, and difficult behavioural issues. Now they lead an almost normal life by following a diet. This does not seem too bad …

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Announcing: Sheila Jones Award

E.S.PKU is very happy to announce the launch of the “Sheila Jones Award”. It’s the first time there is a patient advocate award in the field of PKU and it will be presented at this years annual E.S.PKU conference. If you are interested to learn more about the award and how to submit your entry follow this link: Sheila Jones Award Watch this video for a brief introduction to the award:

PKUDay 2018

International PKU Day is taking place on June 28th. This year once again marks a special year for this important day for Phenylketonuria.  It’s time to celebrate 100th birthday of Horst Bickel. As one of the grounding farther for the PKU diet the E.S.PKU has published the report “The three landmarks of phenylketonuria” once again. You can find it on the official PKUDay website: https://www.pkuday.org/about/pku-day-2018/. Of course you are very keen to learn more about your plans for International PKU Day. Getting it online on the PKUDay website will increase the attention for it – it’s easy – just contact the …

Application deadline for hosting the 2018 ESPKU Annual Conference expired (Updated)

Following the decision of the 2017 ESPKU General Meeting in Hell (Norway), the ESPKU legal members had the opportunity to apply for hosting the 2018 ESPKU Annual Conference until October 31st, 2017. Several applications and proposals have been submitted and will now be evaluated by the ESPKU Executive Board. The final decision will be announced before December 1st, 2017. After careful deliberation the ESPKU Board have awarded the 2018 Conference to Italy. Further details on location and date to follow

Situation of refugee rare metabolic disorder patients (Updated)

According to UNHCR data, currently there are more than 65.000.000 people fleeing from war, conflicts and persecution, as much as never before in history. About 20.000.000 people left their native countries and seek shelter abroad, most of them in Europe. It is a matter of fact that there are a significant number of people amongst these refugees, who suffer from any diagnosed or even undiagnosed rare disease, such as Phenylketonuria (PKU) or other treatable metabolic disorders. All signatory states of the Convention of the United Nations on the rights of persons with disabilities are committed “to take all necessary measures …

European PKU Guidelines Published

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 …

Introducing the “Global PKU Association”

The European Society for Phenylketonuria and Allied Disorders Treated as Phenylketonuria (E.S.PKU) is proud to announce to be part of the foundation of the “Global PKU Association”. You will find further information, like the mission statement in the attachment. As the foundation of this new organization is still in the early phases information will be published over time. For now enjoy reading the official statement below. Global PKU announcement August 1, 2017

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Explaining PKU

Everybody connected to Phenylketonuria (PKU) has been in the situation of explaining what it’s all about. The French association (Les Feux Folleits) has made a great effort to publish a video explaining PKU to children. Enjoy watching, sharing and using this video to explain Phenylketonuria to children. There is also a French version of this video available.  

PKUDay 2017

International PKU Day 2017 is coming up June 28th. If you have planned any activity on or around PKU Day, please let us know on http://www.pkuday.org so we can share the event on the official PKU Day website. You can provide us with an description, image, link and location for your event. It would be great to receive information on your plans soon so we can raise interest in sharing them early! You can download the PKU Day Logo on the PKUDay Website and use it to promote your event. Also if you have questions you can contact us at …

Social media discussion about MipSalus

Recently, a publication on genefo.com about a new treatment in development for Phenylketonuria by Danish biotech company MipSalus, raised considerable discussion on social media. It was suggested that this treatment has already been approved in Europe and awaiting approval in the US. The ESPKU have contacted the CEO of MipSalus for clarification. The statement was published without approval and not accurate. For further information please visit http://www.mipsalus.dk. E.S.PKU has been active to gather information on recent social media topics for you. If you have found something PKU-related that you need clarified, feel free to contact us, we will take care of …