poconnor

🔬 The latest on PKU treatment from NSPKU's October conference - and what they might mean for those living with PKU.

💊 Sepiapterin (Sephience)

🧪 JNT-517, and MZE782

🩸 At-home phe monitors

🗓️ Published on PigPen.page (Want to be the first to see my posts? Subscribe for free emails.)

🔗 https://www.pigpen.page/whats-in-the-treatment-pipeline/

#PKU #Phenylketonuria #RareDisease #InheritedMetabolicDisorders #LivingWithPKU #NSPKU

PKU research update, NSPKU Oct 2025, exciting new treatments

Insights from Prof. MacDonald’s talk at NSPKU 2025 — Sepiapterin, JNT-517, and more — and what they might mean for adults living with PKU.

PigPen: Pauline O'Connor, author & advocate.
Oct 17 at 1:01pmWeb
Show threadSohebOct 17
@poconnor thank you for this update, it was a fascinating read. I'm trying to not get my hopes up too much, but it's quite hard not to.
Show threadpoconnorOct 17

@soheb Thank you, and thank you for the post yesterday :) I did see it and missed doing my own!

Yeah, it is difficult as I worry most about access. A few years back it became clear that the UK wouldn't get the injection.

Now that the UK isn't in the EU, Sepheince isn't a guarantee - particularly for adults.

Re the kidney drugs, None of the UK trials discussed were looking at adults!

I think we and the NSPKU have to gear up for campaigning again as access is going to be a key issue!
#PKU