Hello everyone and welcome to our 2026 Spring Newsletter
Given the recent weird weather changes perhaps this should be renamed the ‘all-seasons’ newsletter! Despite the setbacks with trying to make Procysbi available in the whole of the UK we are still trying to make this happen. We’ve had really lovely surprise of an unexpected donation from the Dyers Company and we have not one, but two new trustees to introduce to you. Have you booked for the CNE International Conference in Dublin in July? If cost is an issue and holding you back then see the article below for possible financial support.
We hope you enjoy the newsletter.
– Will Newman, Chair of the Trustees
CNE International Conference in Dublin 3-4 July 2026
Are you one of the 170 people already booked for the CNE Conference in Dublin on the 3rd and 4th July?
If you’re thinking of going but not sure whether it’s for you, then have a look at our report on the conference in Manchester in 2024 and see what it meant for the people who attended: https://www.cystinosis.org.uk/events/watch-back-elements-of-our-cne-international-conference-2024-better-together/.
If you would like to go but are struggling financially, then please get in touch. CFUK has limited grants to help individuals and families. advice@cystinosis.org.uk
Procysbi in England
We were all really disappointed that the progress to get Procysbi approved for routine prescription in England had been derailed by the abolition of NHS England and the uncertainty regarding the CPAG (Clinical Priorities Advisory Group) process.
Metabolic Support were invited to present to the All Party Parliamentary Group for Rare Genetic and Undiagnosed Conditions in early Feb. They used Procysbi as an example to highlight delays and inequity in getting treatments approved. The following was read out to the meeting:
‘On behalf of the Cystinosis community, supported by the Cystinosis Foundation UK, Metabolic Support and Kidney Research UK, I want to highlight the case of Procysbi, a delayed-release form of cysteamine for people living with nephropathic cystinosis, a life-long, life-limiting rare condition usually diagnosed in early childhood. While clinically similar to existing treatment, Procysbi can be taken every 12 hours rather than every six, removing night-time dosing and medication during the school day, which makes a substantial difference to adherence, sleep, mental wellbeing and daily life for patients and families.
The key challenge we face is blockages in alternative routes to access: because a treatment already exists, Procysbi is not being appraised by NICE, leaving the Clinical Priorities Advisory Group as the only route in England. Despite extensive patient evidence and long delays (15 months thus far), there is still no clear timeline for a decision, and with NHS England being abolished, families are facing growing uncertainty and inequity compared with access in other UK nations.
We ask for:
- Clear timelines and transparency for CPAG decision-making on Procysbi.
- Assurance that the abolition of NHS England will not result in further delay or loss of accountability.
- Action to address inequitable access so people with cystinosis in England are not disadvantaged by where they live.’
We are continuing to work with Chiesi, Metabolic Support, Kidney Research UK and Genetic Alliance to see how we can get Procysbi approved in England and enable equity of access.
Meanwhile in Scotland…..
We are pleased to confirm that Procysbi has officially arrived in Scotland. Trustee, Alex Hutchison, reports that Procysbi is now in her fridge!
Stem Cell Gene Therapy
Do you want to find out more about the cystinosis stem cell trial conducted by Stephanie Cherqui? ‘Last Week in Medicine’ is a weekly podcast co hosted by US doctor, Stephen Jenkins. Stephen is a father of two teenage boys with cystinosis and also a board member of Cystinosis Research Foundation. If you were at the Manchester conference in 2024 you may also remember Stephen’s very clear explanation of cystinosis on the opening day.
In this episode Stephen talks to Dr Stephanie Cherqui about the background to the recent article published in the New England Journal of Medicine, Hematopoietic Stem-Cell Gene Therapy for Cystinosis which updates us on the gene therapy trial in medical/scientific language (https://www.nejm.org/doi/full/10.1056/NEJMoa2506431) You can read the article here and/or listen to the podcast here https://podcasts.apple.com/us/podcast/hematopoietic-stem-cell-gene-therapy-for-cystinosis/id1487590478?i=1000752893799.
We think the real benefit of the podcast is that Stephen Jenkins is able to clarify many of the points to make them more understandable for the lay listener – even though his podcasts are aimed at medical professionals.
Putting the ‘Fun’ in Fundraising
Fun Quiz raises £547 for CFUK and a lot of laughs
On March 14 Margaret and Will Newman ran a Fun Quiz at the local WI Hall. Chinley and Buxworth WI have already nominated CFUK as their chosen charity for the year and this was an extra event. Packing 80 people into the hall in teams of 4 was quite a feat as was answering some of the questions. The raffle grew rapidly in size as people arrived with bottles and boxes of goodies to add to the pile. A fabulous £547 was raised for CFUK. One of the favourite rounds was Norse God or Ikea Furniture which you can try out for yourself here https://take.quiz-maker.com/QLXHCZUS1.
Let us know how you get on!
£3000 Grant donation from The Dyers Company Charitable Trust
hanks to a family connection with our trustee, Tom Kelly, we received the news that The Dyers’ Company Charitable Trust had awarded CFUK a donation of £3000.
Each year The Dyers’ Company Charitable Trust is proud to support over 150 charities with grants. These donations cover a wide and varied area of charitable activity, with particular emphasis on supporting young people, health and welfare causes. The Charity has long standing relationships with the majority of its beneficiaries. Court members and members of the Livery carry out voluntary work and monitor the work of the supported charities, and are able to give feedback on the effectiveness of the charities in fulfilling their objectives.
We are so grateful for this grant and now have a link with a company with roots that stretch back over 550 years – including supporting Swan Upping! You can find out more about the company here https://www.dyerscompany.co.uk/
CFUK welcomes two new trustees
We are delighted to welcome two new trustees to our board and thank them for volunteering their time and expertise. They introduce themselves below but we will be inviting Kevin and Bradley to tell us a bit more about themselves in our Meet the Trustees section of a future newsletter.
Kevin Burgess
I am a dedicated medic and passionate advocate for the cystinosis community. Through my involvement with the Alder Hey Kidney Fund, I work to support children and families facing kidney disease. As a father to a daughter living with cystinosis, my mission is deeply personal. I am committed to making a meaningful difference in the lives of both children and adults affected by cystinosis—through awareness, support, and action.
Bradley Pearson
I am a Project Engineering Manager, working in the water sector based in Manchester. My son was diagnosed with Cystinosis in 2022 at 18 months. Since diagnosis, the foundation has been a great source of information and guidance, especially hosting the CNE International Conference in July 24. I am delighted to be joining as treasurer and trustee to contribute to continue the good work of CFUK for many years to come.
We need you!
Do you have anything you would like to share with the Cystinosis community? Have you a story to offer, a tip to share, a photo to inspire, a fundraising idea to motivate?
Email us at advice@cystinosis.org.uk – we’d love to include more of your stories, tips, etc. in future newsletters.