What question did this study set out to answer?

To review the clinical characteristics, current standard-of-care treatments, and emerging pipeline therapeutics for managing Fabry disease.

May 1, 2025BioDrugsOpen Access

Progress and Challenges in the Treatment of Fabry Disease

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Authors

MLMalte Lenders EMElise Raphaela Menke EBEva Brand

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Overview

Narrative review outlines current enzyme and chaperone therapies alongside emerging pipelines in Fabry disease, highlighting multimodal approaches to mitigate life-threatening organ failure.

Key Points

To review the clinical characteristics, current standard-of-care treatments, and emerging pipeline therapeutics for managing Fabry disease.
Synthesized literature on the pathophysiology and diagnostic workflows for Fabry disease, including leukocyte enzymatic assays and genetic testing.
Evaluated clinical profiles of approved intravenous enzyme replacement therapies and oral pharmacological chaperone therapy.
Surveyed preclinical and clinical-stage developmental pipelines, including gene transfer, messenger RNA, and substrate reduction therapies.
Untreated disease reduces life expectancy by approximately 10 years in female individuals and 20 years in male individuals due to progressive cardiac, renal, and cerebrovascular pathology.
Established standard care consists of biweekly intravenous enzyme replacement therapy (agalsidase alfa, agalsidase beta, or pegunigalsidase alfa) or alternate-day oral migalastat for amenable variants.
Investigational therapies such as gene therapy, substrate reduction therapy, and vesicle-packaged enzyme delivery target current limitations in biodistribution and dosing frequency.

Cite This Study

Lenders et al. (2025) studied this question.

synapsesocial.com/papers/69deac90499d77a496b0c770 https://doi.org/10.1007/s40259-025-00723-3

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May 1, 2025BioDrugsOpen Access

Progress and Challenges in the Treatment of Fabry Disease

View Full Paper

Ask AI

Bookmark

Authors

MLMalte Lenders EMElise Raphaela Menke EBEva Brand

Discussion

Member takes

Overview

Narrative review outlines current enzyme and chaperone therapies alongside emerging pipelines in Fabry disease, highlighting multimodal approaches to mitigate life-threatening organ failure.

Key Points

To review the clinical characteristics, current standard-of-care treatments, and emerging pipeline therapeutics for managing Fabry disease.
Synthesized literature on the pathophysiology and diagnostic workflows for Fabry disease, including leukocyte enzymatic assays and genetic testing.
Evaluated clinical profiles of approved intravenous enzyme replacement therapies and oral pharmacological chaperone therapy.
Surveyed preclinical and clinical-stage developmental pipelines, including gene transfer, messenger RNA, and substrate reduction therapies.
Untreated disease reduces life expectancy by approximately 10 years in female individuals and 20 years in male individuals due to progressive cardiac, renal, and cerebrovascular pathology.
Established standard care consists of biweekly intravenous enzyme replacement therapy (agalsidase alfa, agalsidase beta, or pegunigalsidase alfa) or alternate-day oral migalastat for amenable variants.
Investigational therapies such as gene therapy, substrate reduction therapy, and vesicle-packaged enzyme delivery target current limitations in biodistribution and dosing frequency.

Cite This Study

Lenders et al. (2025) studied this question.

synapsesocial.com/papers/69deac90499d77a496b0c770 https://doi.org/10.1007/s40259-025-00723-3

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