News

Our chair, Will Newman, once again had the opportunity to get involved with the annual Student Voice Prize – an annual, international essay competition that raises the profile of rare diseases within the medical field, particularly with medical students, nurses and scientists who may have never come across rare diseases in their training. Will held virtual meetings with […]

Cystinosis Network Europe and Cystinosis Foundation UK were delighted to host the 2024 International Conference in Manchester, UK in July 2024! 

This family conference was attended by delegates from across the globe, bringing together patients, families, clinicians, researchers, practitioners and pharmaceutical representatives. A diverse range of activities were curated into a packed three day programme, including; a scientific meeting, a range of presentations from world leading experts, live translation into seven languages, a complimentary creche facility, outings for children and young people, an adult patient get together and all topped off with a fun filled hoolie, featuring Morris dancing by known other than our charity Chair, Will Newman!

All of our record-able speaker sessions are now available to view and share. Please see our separate News post to see the list of recordings and related links.

We have had such warm and positive feedback about the whole event from a variety of people that attended. It means the world to us that people truly did feel ‘better together’ by coming along.

Our joint International Conference with CNE, held in Manchester in July 2024, was packed full of brilliant presentations by a range of contributors. We are delighted to be able to share with you a range of recorded outputs from what was an incredible event which truly did help us all feel ‘Better Together’. Watch our […]

We are happy to announce the results of our latest collaborative research, “The impact of different cysteamine formulations on the lives of people living with cystinosis.” This research announces important findings from a survey conducted in collaboration with Metabolic Support UK and Kidney Research UK, aiming to better understand the day-to-day experiences of those living […]

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 […]