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2003;143(4 Suppl):S1-8
Cystinosin-LKG, the alternatively spliced isoform of cystinosin, has a broader localization that could be leveraged to reduce cystine accumulation, restore lysosomal function, and lower apoptosis rates in cystinotic proximal tubule cells [155]
Because GH resistance is common at this stage, IGF-1 may not rise substantially even with adequate GH secretion, so do not use IGF-1 alone to justify dose escalation
Keeping the barrier healthy while introducing new actives is always smart strategy
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