ArticleFrontiers in pediatrics2026
Clinical outcomes of agalsidase Beta (fabrazyme) in Chinese fabry disease patients with proteinuria: a case series.
Article in Frontiers in pediatrics, 2026. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Not yet cited in PubMed.
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Abstract
Background: Fabry disease (FD) is a rare X-linked lysosomal storage disorder caused by pathogenic Cases presentation: Two Chinese male patients with classic FD phenotypes and proteinuria were treated with agalsidase beta (1 mg/kg every two weeks). Case 1 (last followed up at age 34, Conclusions: In these two cases, agalsidase beta was well-tolerated and associated with stabilization of renal and cardiac function. The management of proteinuria required concomitant RAAS inhibition. Family cascade screening remains critical for early diagnosis.
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