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04/04/2025

Now enrolling: Phase I/II CYStem clinical trial

Novartis has announced the opening of its Phase I/II CYStem clinical trial, recruiting children ages 2-5 with nephropathic cystinosis. The trial will evaluate the safety and efficacy of their cellular gene therapy treatment. For more information via the clinical trials website click here. You can also learn more about the study through Novartis’ community statement here.

Research, News 16/09/2026

Cystinosis Ireland announces research valued at €200,000 co-funded with Health Research Board 2026

24/07/2026

CYStem clinical trial community update

Research, News 26/01/2026

Research Update: Development Of The First Human Cellular Model To Study Cystinosis Myopathy