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West et al. Rare Dis Orphan Drugs J 2024;3:22 https://dx.doi.org/10.20517/rdodj.2023.61 Page 27 of 34

by neutralizing anti-drug antibodies in some males. Patients generally fare well with all dialysis modalities
and following a kidney transplant. Emerging treatments include oral substrate reduction inhibitors,
modified ERT, plus gene and T and B cell therapies. Recognition that many aspects of Fabry disease do not
respond to current therapy will drive more research to find adjunctive treatments.

DECLARATIONS
Authors’ contributions
Design, writing, and review of this work: West ML, Geldenhuys L, Bichet DG

Availability of data and materials
Not applicable.

Financial support and sponsorship
None.

Conflicts of interest
West ML has received research funding, speaker’s fees and/or consultant fees from the following: Alexion,
Amicus, Chiesi, Idorsia, Protalix, Sanofi, Sumitomo, and Takeda; he shares IP in Fabry gene therapy and
Fabry cardiac biomarkers. Bichet DG has received research funding, speaker’s fees and/or consultant fees
from Amicus Therapeutics, Sanofi, and Takeda. Geldenhuys L declares no conflicts of interest.

Ethical approval and consent to participate
Not applicable.

Consent for publication
Not applicable.

Copyright
© The Authors 2024.

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