Article

Aligning Fabry-specific treatment access with contemporary evidence: Considerations for the Australian setting

2022-08-25

Abstract excerpt

<title>Abstract</title> <p><bold>Background</bold>: Fabry disease is a rare, progressive, X-linked, lysosomal storage disorder characterized by a wide spectrum of heterogeneously progressive clinical phenotypes. In the absence of a cure, disease-specific therapy aims to improve symptoms, stabilize current disease and delay progression. In Australia access to available treatments requires patients to meet pre-spec...

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Literature Corpus work: 2228262a-7b0f-5dc5-89e8-284b94e91e20
DOI: 10.21203/rs.3.rs-1965266/v1

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