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Health‐related quality of life and patient‐reported outcome measurements in patients with cystinosis

Nephropathic cystinosis is a rare autosomal recessive lysosomal storage disorder. With the availability of treatment and renal replacement therapy, nephropathic cystinosis has evolved from an early fatal disease to a chronic, progressive disorder with potentially high impairment. We aim to review th...

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Detalles Bibliográficos
Autores principales:	Witt, Stefanie, Kristensen, Kaja, Hohenfellner, Katharina, Quitmann, Julia
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	John Wiley & Sons, Inc. 2022
Materias:	Research Reports
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9981409/ https://www.ncbi.nlm.nih.gov/pubmed/36873093 http://dx.doi.org/10.1002/jmd2.12352

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