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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 26/01/2026

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

Research 26/01/2026

Research update

News, Research 23/10/2025

CF10 Clinical Trial Update