Conferences
The fight for sapropterin means the NSPKU team now know a great deal about the licensing process. This bodes well for future campaigns. As does the experience, which shows that the advocacy of patients tipped the balance.
Advocacy
One of the consistent highlights of NSPKU conferences over the years has been the patient panel. This year, the panel was called PKU United. Because we are stronger as a united community.
Pauline O'Connor (PigPen) articles on PKU, Brain Injury, mental health, Phenylketonuria, concussion, TBI, ABI, Rare Disease
Conferences
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”.
Conferences
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).
News
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.
News
- A brain injury manifesto for the UK election - Two research opportunities - Share your views on medicine shortages. - And tell the government what you think of PIP
News
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?
News
The NSPKU conference held from 17-19 May 2024 included an update on possible new treatments for PKU. These are my notes from the sessions on sepiapterin, mRNA, gene therapy, solute carrier 6, and options for PegPal.
News
This research study is looking at ways to improve mood after brain injury by taking part in rewarding activities
News
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...
News
This short story anthology is my first fiction book, and the choice to try a Kickstarter was strategic. The Kickstarter campaign successfully funded! Grab your copy now