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Burosumab treatment for FGF23-related hypophosphatemia in a two-year-old girl with McCune-Albright syndrome

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McCune-Albright syndrome (MAS) is a rare mosaic disorder caused by a gain-of-function pathogenic GNAS variant that triggers endocrine and skeletal manifestations, including fibrous dysplasia (FD) and FGF23-related hypophosphatemia. Conventional treatments (e.g... ...