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… Cystagon / Procysbi Survey

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)… QUALIFY-US Study

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… Novartis Gene Therapy Update

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… Contact: For Families With Disabled Children

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… Gene Therapy Trials Update

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… History of Cystinosis – UK Disability History Month

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… Procysbi Available in Wales

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… Marathon Man Raises £1134

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… Our Experience with the Student Voice Prize

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… Eye Drops Supply Issues