Clinical outcomes of agalsidase Beta (fabrazyme) in Chinese fabry disease patients with proteinuria: a case series - Takeaways - MDSpire

Clinical outcomes of agalsidase Beta (fabrazyme) in Chinese fabry disease patients with proteinuria: a case series

  • By

  • Zelei He

  • Yuanyuan Wu

  • Hong Yang

  • Yijuan Li

  • Ling Xu

  • June 17, 2026

  • 0 min

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  • 1

    Fabry disease is an X-linked lysosomal storage disorder caused by GLA gene mutations, leading to α-galactosidase A deficiency.

  • 2

    Agalsidase beta (Fabrazyme) is approved in China for enzyme replacement therapy in Fabry disease patients.

  • 3

    Two Chinese male patients with Fabry disease showed improved renal function and reduced proteinuria after treatment with agalsidase beta.

  • 4

    Both patients tolerated agalsidase beta well, experiencing symptom regression without infusion-related adverse events.

  • 5

    Concomitant RAAS inhibition was necessary for effective management of proteinuria in these patients.

Original Source(s)

Clinical outcomes of agalsidase Beta (fabrazyme) in Chinese fabry disease patients with proteinuria: a case series

  • by Zelei He, Yuanyuan Wu, Hong Yang, Yijuan Li, Ling Xu

  • June 17, 2026

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