This is a questionnaire for everyone with PKU or their carers. We want to know if you have been tested with sapropterin or not. What is your experience? We look forward to hearing from you nspku.org/survey-share-y…
Some very interesting early results with the new drug Repinatrabit (JNT 517) in 4 adolescents with PKU showing that blood Phe decreased from 1000 to target ranges after 4 weeks of an open label study
Thank you to my team for making tonight very special. Ten years ago today I tried to retire - but somehow - we are sill here! The slippers remain in their box!
I keep thinking about this in Birmingham. One person can make a huge difference in so many lives. This Easter event for the kids was all thanks to someone working tirelessly behind the scenes. We were lucky to help a little and experience the joy of being together 🙏🏼
@macdonj
Two-Year Outcomes of Sapropterin Treatment in Children with Phenylketonuria: A Longitudinal Observational Study of Metabolic, Dietary, and Psychosocial Effects mdpi.com/3703528#mdpinutrients via @Nutrients_MDPI
WE NEED THE RESEARCH BUT I NEED YOU...
For the next study of my PhD to take place and get this much needed research, I need YOU! If you have PKU, are aged 18 or over (UK based) and do regular activity please give me a message or email Annabelle.Skidmore@mail.bcu.ac.uk Thank you!