We need YOU to write to your MP asking for their support with our Parliamentary campaign for fair treatment for PKU.

Not sure how to explain PKU? This template letter can help you write to your MP – you can copy it and adapt it 0 make sure you full in the sections about how PKU affects you.

It is best to use your own words as much as possible, especially when you explain how PKU impacts you or your family. You will need to include your full postal address with post code. It is OK to write to your MP by email.

If you are not sure who your MP is, or their email address, you can find out here https://members.parliament.uk/FindYourMP

[Your Name]
[Your Address]
[Postcode]
[Email Address]
[Date]

Dear [MP Name],

Re: Support for people living with Phenylketonuria (PKU) and fair access to new treatments

I am writing to you as one of your constituents to ask for your support for people living with Phenylketonuria (PKU), a rare inherited metabolic condition, and to help ensure that patients in the UK are not left behind as new treatments become available internationally.

PKU is a lifelong genetic condition that prevents the body from properly breaking down the amino acid phenylalanine. Without treatment, phenylalanine builds up in the blood and can cause serious and irreversible damage to the brain and nervous system.

People with PKU must follow a highly restrictive low-protein diet for life, supported by specially manufactured protein substitutes and low-protein foods. The condition affects every aspect of daily life, including education, employment, family life, social activities and mental wellbeing. Even with excellent dietary management, many people continue to experience significant challenges.

[Please describe your own experiences and concerns. Tell your own story!]

Explain if you have PKU or your family member has PKU. You may wish to include:

• The daily burden of managing PKU – e.g my child can only 4g protein a day, that is equivalent to one slice of bread.
• The impact on education, work, family life or mental health – e.g I struggle with the diet, it is so restrictive and complicated and my phenylalanine levels are not well controlled.
• Challenges accessing treatment and support – eg I struggle to get the food I need from my chemist.
• Concerns about the future – e.g. my child’s diet is so restrictive I worry they will rebel against it when they get older.
• The impact on carers and family members – e.g. managing my child’s PKU is a stress for me and my other children.

I am writing now because there is growing hope for the PKU community through the development of new treatments.

One such treatment is sepiapterin, which is being introduced in parts of Europe and the United States. Sepiapterin has been trialled in Birmingham Children’s Hospital and was found to be very effective, allowing users to eat a normal diet with well controlled PKU. The license holder, PTC Therapeutics has promised to seek a UK license later this year. While the UK licensing and assessment process is separate, many people with PKU are concerned that patients here could once again face lengthy delays in accessing innovative treatments compared with people living in other countries.

Unfortunately, the UK has a mixed record when it comes to providing timely access to medicines for PKU; the medicine Kuvan (sapropterin) was not made available here for 12 years after licensing and the treatment pegvaliase was never licensed in the UK. There is understandable concern within the PKU community that history may repeat itself.

At the same time, scientific research into PKU is advancing rapidly. Sepiapterin is only one example of several promising developments that could significantly improve health outcomes and quality of life for people living with the condition. We want to ensure that the UK remains committed to providing fair and equitable access to innovation for people with rare diseases.

In previous years, parliamentarians supported people with PKU through an All-Party Parliamentary Group (APPG) focused on PKU and inherited metabolic disorders. While that group is no longer active, there is now an opportunity to rebuild parliamentary engagement on PKU.

This could take the form of a re-established APPG, or alternatively a more informal cross-party group of interested MPs and peers working together to raise awareness of PKU, understand the challenges faced by patients and families, and champion fair access to treatment.

I understand that Liz Twist MP and Jeremy Hunt MP have already agreed to support efforts to raise awareness of PKU and the needs of the PKU community within Parliament. I hope you would consider joining these efforts.

I would be grateful if you could:

1. Meet with me to learn more about the realities of living with PKU and the challenges faced by people affected by the condition.
2. Support parliamentary efforts to improve awareness and understanding of PKU.
3. Work with other interested MPs, including Liz Twist MP and Jeremy Hunt MP, to explore the re-establishment of a parliamentary group focused on PKU, or the creation of a new cross-party network of parliamentarians with an interest in PKU.
4. Advocate for fair, timely and equitable access to current and future PKU treatments.
5. Engage with ministers, NHS bodies and other decision-makers to ensure that people with PKU are not left behind as new therapies become available internationally.

Although PKU is a rare condition, its impact on those living with it is profound. New scientific advances offer real hope to patients and families, but only if people in the UK are able to benefit from them.

I would be very grateful for your support and would welcome the opportunity to discuss this issue with you further.

Thank you for taking the time to consider my concerns. I look forward to hearing from you.

Yours sincerely,

[Your Name]

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