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The renal Fanconi syndrome in cystinosis: pathogenic insights and therapeutic perspectives
Nature Reviews Nephrology, 2016Stephanie Cherqui
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Cysteamine therapy delays the progression of nephropathic cystinosis in late adolescents and adults
Kidney International, 2012Chris Ottolenghi, Pierre Cochat
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Nephropathic cystinosis: an international consensus document
Nephrology Dialysis Transplantation, 2014Stephanie Cherqui+2 more
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Hematopoietic Stem Cell Gene Therapy for the Multisystemic Lysosomal Storage Disorder Cystinosis
Molecular Therapy, 2013Celine J Rocca+2 more
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