Cystinosis Clinical Trial
Feb 02, 2026
Cystinosis Treatment: Transforming a Life-Limiting Disorder into a Manageable Chronic Condition
Summary Approximately 1,600 prevalent cases were diagnosed across the 7MM in 2025, underscoring the condition's rarity and the concentration of patients at specialized metabolic and nephrology centers. Treatment is limited to long-standing cysteamine products, PROCYSBI, CYSTAGON/NICYSTAGON (cysteamine bitart...
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