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Graphical abstract Similar content being viewed by others Background Nephropathic cystinosis is a rare, autosomal recessive lysosomal storage disease with an incidence of 1:100,000 to 1:200,000 live births [1]

Experts caution that real-world effectiveness will depend on whether patients can maintain daily dosing and timing requirements over the long term

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Pending the accumulation of adequate clinical evidence, these extracts should not be recommended as equivalent to verified functional factors