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

Abstract Background Nephropathic cystinosis is a rare and severe metabolic disease leading to an accumulation of cystine in lysosomes which especially harms kidney function. A lifelong therapy with the aminothiol cysteamine can delay the development of end-stage renal disease and the necessity of ki...

Full description

Bibliographic Details
Main Authors: Christina van Stein, Sabrina Klank, Marianne Grüneberg, Chris Ottolenghi, Jürgen Grebe, Janine Reunert, Erik Harms, Thorsten Marquardt
Format: Article
Published: BMC 2021-09-01
Series:Orphanet Journal of Rare Diseases
Online Access:https://doi.org/10.1186/s13023-021-01991-2