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Monthly medication cost is $375, with ongoing follow-up to track progress, adjust dosing, and support lifestyle changes

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Age, body weight, baseline metabolism, and stomach sensitivity all contribute to side-effect timing and intensity

2 , 31IN32 (2009)

202,203 Essentially, the imported disulphide-containing proteins undergo cathepsin-catalysed degradation in the lysosome, leading to the formation of cystine, which is then effluxed by the cystinosin transporter into the cytosol, where it is reduced to cysteine in a process that requires NADPH, leading to the formation of oxidised glutathione

Its not doing anything, said Frankl