Low Protein

Lived Experiences of Sapropterin

An NSPKU presentation on a forthcoming PhD “to collect information about the rollout and response testing for sapropterin treatment in England since its availability on the NHS for all ages in 2021”.

#PKUDay2024

Exciting new research and clinical trials in the UK, which you can support for PKU Day 2024. June 28 is a chance for this rare disease community to raise awareness of the inherited metabolic condition, Phenylketonuria (PKU).

Review of the EU Guidelines on PKU

The guidelines...help inform policy changes and establish the best treatment practices in places where those are still being developed, and to support those in areas where treatment falls short.

PKU News, June 2024

Update on sepiapterin and other groundbreaking treatments. A primer on the PKU injection treatment PegPal, AKA Pegvaliase or Palynziq. A new way to distribute treatments for Rare Disease?

PKU news May 2024

Looking for a massive online resource with recipe & product ideas, managing PKU and fitness, and honesty about living with PKU? Check out Tristan’s blog...

Starting the Conversation

We wanted a space where adults with PKU felt able to hold an open discussion about managing PKU, and to collect data about our concerns in real time.

Emergency travel with PKU

People with PKU need to prepare for the unexpected, here are my tips for travel. What are yours?

Tips for blood spots

TW: discusses blood and needles. Not all lancets are equal! While no blood collection method is fun or painless, the type of lancet used does have a significant effect on pain and healing.

Sapropterin trial, part 3: the trial

The blood spots from my first week on the pills seemed to show a drop. I admit that I began to get excited. That was a mistake, and one I’d encourage others to avoid.

Sapropterin trial, part 2: Baseline testing

Maintaining this level of control would be a tough ask. But I discovered that, when I thought I had been strict on my diet in the past, I really hadn’t.