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

Autor: Maria Helena Vaisbich, Juliana Caires Ferreira, Heather Price, Kyleen D. Young, Saba Sile, Gregg Checani, Craig B. Langman

Publikováno v: JIMD Reports, Vol 63, Iss 1, Pp 66-79 (2022)

Abstract 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 child

Externí odkaz: https://doaj.org/article/d94e9d5fb4c941ffa28be53443ea2b05

Off label uses of pamidronate in rare pediatric bone diseases (Jansen's Metaphyseal Chondrodysplasia and Generalized Arterial Calcification of Infancy): A four year perspective

Autor: Kyleen D. Young, Craig B. Langman, Harald Jueppner

Publikováno v: Bone Abstracts.

Externí odkaz: https://explore.openaire.eu/search/publication?articleId=doi_________::c6988af9a7f51bf701dff249f0a551c9
https://doi.org/10.1530/boneabs.7.p130

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.

Autor: Vaisbich MH; São Paulo University Medical School São Paulo Brazil., Caires Ferreira J; Instituto da Criança do Hospital das Clínicas da Faculdade de Medicina da Universidade de São Paulo (HCFMUSP) São Paulo Brazil., Price H; Ann & Robert H. Lurie Children's Hospital of Chicago Chicago Illinois USA., Young KD; Ann & Robert H. Lurie Children's Hospital of Chicago Chicago Illinois USA., Sile S; Horizon Therapeutics plc Deerfield Illinois USA., Checani G; Horizon Therapeutics plc Deerfield Illinois USA., Langman CB; Feinberg School of Medicine Northwestern University Chicago Illinois USA.

Publikováno v: JIMD reports [JIMD Rep] 2021 Nov 01; Vol. 63 (1), pp. 66-79. Date of Electronic Publication: 2021 Nov 01 (Print Publication: 2022).