A comparison of immediate release and delayed release cysteamine in 17 patients with nephropathic cystinosis

van Stein, C; Klank, S; Gruneberg, M; Ottolenghi, C; Grebe, J; Reunert, J; Harms, E; Marquardt, T

van Stein, C; Marquardt, T (corresponding author), Univ Munster, Dept Gen Pediat, Metab Dis, Albert Schweitzer Campus 1, D-48149 Munster, Germany.

ORPHANET JOURNAL OF RARE DISEASES, 2021; 16 (1):

Abstract

Background Nephropathic cystinosis is a rare and severe metabolic disease leading to an accumulation of cystine in lysosomes which especially harms ki......

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