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

Over the last decade, access to investigations (namely genetics and IL-CL) and to cysteamine have improved in DEing and TrE. However, discrepancies remain with DEed: access to delayed released cysteamine is limited, and reimbursement is still profoundly insufficient, therefore limiting their current use. A higher resolution version of the Graphical abstract is available as Supplementary information.