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Moreover, only 54% of patients in those economics have access to oral cysteamine therapy, and there is limited access to cysteamine eye drops, supportive therapies, and standard diagnostic and monitoring tools, all of which are essential for the prevention of disease progression and extended lifespan [159]
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Biochemical Journal
TaddeiCZhouBBixbyHCarrillo-LarcoRMDanaeiGJacksonRTet al
doi:10.1016/j.yexcr.2019.05.032 255
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