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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.
Cystinosis Ireland announces research valued at €200,000 co-funded with Health Research Board 2026
CYStem clinical trial community update
Research Update: Development Of The First Human Cellular Model To Study Cystinosis Myopathy