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-Reference-Cited by-同舟云学术

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

Published:2021-11 Issue:1 Volume:63 Page:66-79
ISSN:2192-8312
Container-title:JIMD Reports
language:en
Short-container-title:JIMD Reports

Author:

Vaisbich Maria Helena¹^ORCID,Caires Ferreira Juliana²^ORCID,Price Heather³,Young Kyleen D.³,Sile Saba⁴,Checani Gregg⁴,Langman Craig B.⁵^ORCID

Affiliation:

1. São Paulo University Medical School São Paulo Brazil

2. Instituto da Criança do Hospital das Clínicas da Faculdade de Medicina da Universidade de São Paulo (HCFMUSP) São Paulo Brazil

3. Ann & Robert H. Lurie Children's Hospital of Chicago Chicago Illinois USA

4. Horizon Therapeutics plc Deerfield Illinois USA

5. Feinberg School of Medicine Northwestern University Chicago Illinois USA

Publisher

Wiley

Subject

Biochemistry, Genetics and Molecular Biology (miscellaneous),Endocrinology, Diabetes and Metabolism,Internal Medicine

Link

https://onlinelibrary.wiley.com/doi/pdf/10.1002/jmd2.12260

Reference41 articles.

1. Cystinosis: a review

2. The History of Cystinosis: Lessons for Clinical Management

3. Cystinosis: clinical presentation, pathogenesis and treatment;Ivanova E;Pediatr Endocrinol Rev Suppl,2014

4. Nephropathic Cystinosis: Symptoms, Treatment, and Perspectives of a Systemic Disease

5. Growth retardation in children with cystinosis;Besouw M;Minerva Pediatr,2010

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1. Cystinosis — a review of disease pathogenesis, management, and future treatment options;Journal of Rare Diseases;2024-06-10

2. Switching from immediate- to extended-release cysteamine in patients with nephropathic cystinosis: from clinical trials to clinical practice;Clinical Kidney Journal;2024-03-06

3. Gastrointestinal challenges in nephropathic cystinosis: clinical perspectives;Pediatric Nephrology;2024-02-23

4. Enteric-Coated Cysteamine Bitartrate in Cystinosis Patients;Pharmaceutics;2023-06-29

5. Long-term clinical benefits of delayed-release cysteamine bitartrate capsules in patients with nephropathic cystinosis (response to “A comparison of immediate release and delayed release cysteamine in 17 patients with nephropathic cystinosis”);Orphanet Journal of Rare Diseases;2023-06-23