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Worldwide disparities in access to treatment and investigations for nephropathic cystinosis: a 2023 perspective

Nephropathic cystinosis (NC) is a rare lysosomal disease, leading to early kidney failure and extra-renal comorbidities. Its prognosis strongly relies on early diagnosis and treatment by cysteamine. Developing economies (DEing) face many challenges when treating patients for rare and chronic disease...

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Main Authors: Regnier, Maitena (Author) , Flammier, Sacha (Author) , Boutaba, Mounia (Author) , Ndongo, Aliou Abdoulaye (Author) , Servais, Aude (Author) , Schaefer, Franz (Author) , Levtchenko, Elena (Author) , Bacchetta, Justine (Author) , Bertholet-Thomas, Aurélia (Author)
Format: Article (Journal)
Language:English
Published: April 2024
In: Pediatric nephrology
Year: 2024, Volume: 39, Issue: 4, Pages: 1113-1123
ISSN:1432-198X
DOI:10.1007/s00467-023-06179-3
Online Access:Verlag, kostenfrei, Volltext: https://doi.org/10.1007/s00467-023-06179-3
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Author Notes:Maitena Regnier, Sacha Flammier, Mounia Boutaba, Aliou Abdoulaye Ndongo, Aude Servais, Franz Schaefer, Elena Levtchenko, Justine Bacchetta, Aurélia Bertholet-Thomas
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Summary:Nephropathic cystinosis (NC) is a rare lysosomal disease, leading to early kidney failure and extra-renal comorbidities. Its prognosis strongly relies on early diagnosis and treatment by cysteamine. Developing economies (DEing) face many challenges when treating patients for rare and chronic diseases. The aim here is to evaluate the access to investigations and treatment in DEing, and to assess for potential inequalities with Developed Economies (DEed).
Item Description:Online veröffentlicht am 18. November 2023
Gesehen am 11.03.2024
Physical Description:Online Resource
ISSN:1432-198X
DOI:10.1007/s00467-023-06179-3

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