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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]
There is a great need to develop experimental strategies to characterize the glutathione metabolism upon consumption (both synthetic and natural) in vivo
A key figure working in this area was James Gowans, a British physician and immunologist based at the Sir William Dunn School of Pathology in Oxford
It requires weight-based dosing and IV or SubQ administration under clinical supervision
Can injectables be combined with other therapies
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