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A curling stone and two sweepers

Scottish Medicines Consortium Approves Procysbi for NHSScotland Use, Offering New Hope and Choice for Cystinosis Patients

The Scottish Medicines Consortium (SMC) has today accepted mercaptamine (Procysbi) for use within NHSScotland, following a third resubmission assessed under the orphan equivalent medicine process. This landmark decision marks a pivotal moment for individuals living… Scottish Medicines Consortium Approves Procysbi for NHSScotland Use, Offering New Hope and Choice for Cystinosis Patients

Lena in the lab at RGU

From Patient to PhD: Cystinosis Advocate Begins Doctoral Journey and Reunites with CFUK-Donated Lab Equipment

A remarkable full-circle moment unfolded this autumn at Robert Gordon University (RGU), as adult cystinosis patient, Lena Forsyth, began her PhD in Trauma-Induced Coagulopathy at the School of Pharmacy Applied Sciences and Public Health –… From Patient to PhD: Cystinosis Advocate Begins Doctoral Journey and Reunites with CFUK-Donated Lab Equipment

Cystinosis Foundation UK Celebrates Major Milestone: £3.9 Million Award to Advance Ground Breaking Treatment into Clinical Trials

Cystinosis Foundation UK is proud to announce a momentous step forward in the fight against cystinosis: researchers at the University of Sunderland have received £3.9 million from the Medical Research Council (MRC) to take a… Cystinosis Foundation UK Celebrates Major Milestone: £3.9 Million Award to Advance Ground Breaking Treatment into Clinical Trials

Eloise in a Viking helmet.

Eloise’s story is a candid portrait of childhood with cystinosis—showing how family, honesty, and routine can empower a young girl to thrive.

Diagnosed at 18 months, Eloise lives with cystinosis. Her parents, Jenni and Tom, share the emotional journey from diagnosis to daily management, including the challenges of foul-tasting medication, disrupted sleep, and the importance of early,… Eloise’s story is a candid portrait of childhood with cystinosis—showing how family, honesty, and routine can empower a young girl to thrive.

Talking the Talk

Contributing to the Cambridge School of Clinical Medicine’s Patient Voice programme Our chair, Will, was delighted to receive an invitation to talk to Year 4 Medical Students at Cambridge School of Clinical Medicine in November… Talking the Talk

Lena’s journey from patient to biomedical scientist offers a rare and inspiring glimpse into life with cystinosis

Diagnosed with nephropathic cystinosis at just seven months old, Lena spent years navigating a complex regimen of medications, blood tests, and hospital visits. Her curiosity about what happened to her blood samples led her to… Lena’s journey from patient to biomedical scientist offers a rare and inspiring glimpse into life with cystinosis