Our charity NSPKU has been campaigning in Parliament for understanding and support for PKU since 2017. This started by people living with PKU asking their MPs for help. We need to do this again and everyone can help. Write to your MP in your own words – you can do this in an email or by letter.
Here is a template letter you can use to get you started. The best way to write to your MP is to talk about your real experiences and concerns. Remember to include your full address and postcode.
If you are not sure who your MP is, you can look up their details using this link : https://members.parliament.uk/FindYourMP
My Name
Address
Postcode
My email address :
Dear Mr/Ms [insert the name of your MP]
The rare disease PKU
I am writing to you about the rare disease phenylketonuria (PKU) [which affects me/my son/daughter/family member…] People with PKU cannot metabolise phenylalanine, a component of protein. As the phenylalanine is not broken down it can build up in the brain causing permanent brain damage. Currently, the main treatment funded in the UK is a complex dietary treatment, in which almost all sources of protein are removed from the diet. PKU is diagnosed at birth by the “heel-prick” test.
PKU is important to me because [explain who has PKU in your family and the impact it has. MPs are more likely to help if you explain the personal issues for you.]
The dietary treatment for PKU is very difficult. Often people with PKU can eat only tiny amounts of natural protein – such as in one or two slices of bread. People rely upon synthetic protein substitute and prescription foods. The diet is complex and time-consuming and not everyone copes. Failing to manage phenylalanine levels can have devastating effects on health.
There is a drug treatment for PKU called sapropterin (the branded name is Kuvan) which works for about 30% of people with PKU. The UK was very slow to adopt this drug – the PKU community waited over 10 years to access it. It has been life changing for many people with PKU, but there are concerns about why it is so hard to get treatments for PKU in the UK.
There are also other innovative treatments for PKU. An injectable enzyme treatment for adults (pegvaliase) was licensed in 2019 and is also not available on the NHS. Other new treatments are expected soon.
People with PKU also need access to modern blood testing technology, similar to that used by people with diabetes. At the moment, people with PKU have to post blood samples to hospital laboratories using a method which has not been updated since the 1960s. Modern blood monitoring equipment is under development and will have a huge impact on patient welfare if it is swiftly adopted into use by the NHS.
I really hope that patients in the UK get access to modern treatments and technology because […. Say what it would mean for you/your family member…]
There are also concerns about the provision of basic support for people with PKU – for example access to dietary foods and supplements, psychological support and access to expert metabolic clinics. Patients in the UK suffer patchy care, where the level of support and care received will depend on where you live. Mental health issues associated with PKU or the highly restrictive diet are often not supported well. [Explain any concerns you have personally]
In the last Parliament, a group of MPs formed an APPG chaired by Liz Twist MP. The APPG examined whether the drug appraisal system is working properly for rare diseases like PKU and debated the issue in Parliament. The APPG also looked at wider issues affecting people with PKU. The APPG is supported by the national charity for PKU (NSPKU) and is independent of pharmaceutical companies.
I would be very pleased if you could support my family and other people affected by PKU. You could support the formation of the APPG in the next Parliament.
Yours sincerely
