PKU News, June 2024
A monthly selection of news and events related to Phenylketonuria (PKU).
PKU Unity, the Chair’s notes on the 2024 NSPKU patient panel
I was honoured to be asked to chair the annual patient panel at this year’s NSPKU conference: PKU Unity. This was my introductory speech:
“One of the consistent highlights of NSPKU conferences over the years has been the patient panel. This year, the panel is called PKU United. Because we are stronger as a united community.
We need the PKU community to be a safe place to celebrate, commiserate, and campaign together.
The panel will introduce ourselves shortly, and we are going to spend the next forty minutes reflecting on the fact that people with PKU are now not all on the same treatment. Some may have had a wonderful few years experimenting with new treatments and opportunities. Others may have had a very difficult time discovering that certain new treatments are not available or effective.
We need the PKU community to be a safe place to celebrate, commiserate, and campaign together. The first step is to acknowledge that there are now different treatment experiences within our community. Basically, change is happening.
Hopefully, change will be for the better, that is certainly what the campaigns are about. What is certain is, more than ever, we need to know we are not alone. And that takes work. We need to work on our ties to ensure we can help each other remain a cohesive, supportive and forceful patient community.”
PKU future treatments update & PegPal
The NSPKU conference kicked off with a look at treatments beyond diet and sapropterin. The first two speakers were looking ahead to ‘what is in the pipeline.’ These were fascinating sessions on advances in technology and discoveries in trials which are at the edge of what we know about PKU. I wrote about the sessions on future treatment here.
My previous primer about the ‘PKU injection treatment’ variously known as PegPal, Pegvaliase, or Palynziq may also be of interest.
I will be posting further blogs on the sessions. As some of these involved sensitive data, I’m checking with the presenters before posting publicly.
Problems with prescriptions?
There was plenty of anecdotal discussion regarding shortages of supplements and special foods on prescription at the conference. We need to be able to collate this information before it can be acted on. With perfect timing, the Royal Pharmaceutical Society wants to hear from patients who have been affected by medicine shortages.
They have created an online survey to collect experiences of patients and the impact of shortages. If you’ve had trouble getting your food, be sure to shout about it!
What do you think of PIP?
The government is concerned that too many people will have access to PIP (yes, really!) and has opened a consultation on “the sustainability of the current model”. Plenty of people with PKU have been through this process with varying levels of success. This is your chance to provide feedback in a rather bureaucratic survey. But stick with it, and let’s get our voices heard.
Reading:
This article on medical research will certainly be of interest to PKU patients.
“If we can stop treating people as commodities and instead recognise that transforming patients’ lives is priceless, then we can ensure that more life-saving treatments are reaching those who desperately them.”
