News

Do you have experience of cystagon (mercaptamine), procysbi (cysteamine bitartrate), or both? Take part in our questionnaire to help decision-makers understand the impact cystinosis and its treatment has on the day-to-day quality of life of people living with cystinosis, as well as their parents and carers. Anyone living with or caring for someone with cystinosis […]

Participants are needed for the testing of a newly developed questionnaire to measure the quality of life of patients with cystinosis. The study coordinators are especially looking for parents of younger children (age 0 -7) and of young adults, as well as children and teenagers (age 8-17) who are willing to fill out the questionnaire […]

We have received this message from Novartis regarding their gene therapy program for cystinosis: “Dear Cystinosis Community, In May 2023, Novartis acquired the investigational autologous hematopoietic stem cell (HSC) gene therapy program for the treatment of cystinosis from AVROBIO. We look forward to continuing the development of this program and bringing new hope to patients […]

We’ve recently discovered this organisation that offers a wealth of information, advice and support, for example on subjects such as: Benefits and tax credits; Early years support; Social care; Education and learning. They run a free advice and information helpline parents and carers with a disabled child aged from birth to 25, living in any part of […]

If you are following the gene therapy trials in the USA you will have seen that the 6th and final patient was dosed at the end of October. AVROBIO, the company that is developing the technology, indicate that they are hoping to start their own trials in 2023 and they have also said that they […]

by Ami Froehlich, Trustee 18 November to 18 December marks Disability History Month, with this year’s theme focused on Disability, Health and Well Being. UK Disability History Month (UK DHM) celebrates historical contributions of people with disabilities to society by raising awareness of challenges and structural barriers faced by them. To celebrate this years Disability […]

Following a decision by the ‘All Wales Therapeutics and Toxicology Centre’ Procysbi will now become routinely available in the NHS across Wales to treat cystinosis. We took part in the appraisal process last year (December 2021) in partnership with Metabolic Support UK and we’re very pleased that this is the outcome, you can find out […]

In October last year Tom Wood ran his first ever marathon to raise money for CFUK. Sporting one of our t shirts Tom finished the Manchester Marathon in the commendable time of 3 hrs 53 minutes and raised a tremendous £1134. Tom’s partner’s daughter, Maeve, has cystinosis. Tom posted the following on completion: “I can […]

It’s good to talk – It’s good to listen(Our experience with the Student Voice Prize) One of Cystinosis Foundation UK’s aims is to help inform the medical profession about cystinosis, not only to help with early diagnosis but also to enable patients and families to have a better experience in the healthcare system. When I […]

We recently became aware that many patients have been unable to get any supply of the unlicensed 0.55% cysteamine hydrochloride eye drops they are prescribed. We contacted Guy’s and St Thomas’ NHS Foundation Trust, who manufacture the drops, they told us that they are “currently out of stock of cysteamine hydrochloride 0.55% eye drops following […]