Cysteamine bitartrate delayed‐release capsules control leukocyte cystine levels and promote statural growth and kidney health in an open‐label study of treatment‐naïve patients <6 years of age with nephropathic cystinosis

Nephropathic cystinosis is a rare autosomal recessive lysosomal storage disease that is characterized by accumulation of cysteine and formation of crystals within cells of different organs and tissues causing systemic manifestations in childhood that include poor linear growth, ocular involvement, h...

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Detalles Bibliográficos
Autores principales: Vaisbich, Maria Helena, Caires Ferreira, Juliana, Price, Heather, Young, Kyleen D., Sile, Saba, Checani, Gregg, Langman, Craig B.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: John Wiley & Sons, Inc. 2021
Materias:
Research Reports
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8743345/
https://www.ncbi.nlm.nih.gov/pubmed/35028272
http://dx.doi.org/10.1002/jmd2.12260

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8743345/
https://www.ncbi.nlm.nih.gov/pubmed/35028272
http://dx.doi.org/10.1002/jmd2.12260

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